Good news! GREAT news actually. Trey’s C1 or his top vertebrae is ossifying (turning into bone). In people with MPS, the top vertebrae doesn’t form completely. It is what leads to neck instability. When Trey was diagnosed, we were told his C1/C2 was unstable because it was not hard bone, but more like cartilage. We were also told that build up of GAGs in his neck is causing spinal cord compression. Not severely, but somewhat. This is typical. The combination of spinal cord compression and instability is very dangerous. Paralysis if you fall the wrong way kind of dangerous, meaning no gymnastics, horseback riding, contact sports etc. However, we found out at Trey’s orthopedics appointment this week, that Trey’s C1 is ossifying and that he has NO neck instability. With MPS, we hope for stability, no progression. But for the opposite news, that he is improving? YAHOO! Trey’s MRI showing spinal cord compression was taken before Trey’s first intrathecal dose, as part of the qualifying tests in August 2011, so I am curious to see what the end of study tests show this March.
Opthalmology… last year we found out Trey has some ‘changes’ in his eyes. White spots that cause issues first with night vision, then permanently. However, during my ‘Oh My God my kid is going blind but maybe not if he qualifies for the intrathecal trial’ scare, I called Dr. Muenzer and he said no, these changes do not cause blindness in MPS II. They can cause eventual night vision issues, but not yet in Trey. Last Thursday we found out those spots are still there, but still not causing any vision issues. Trey still has 20/20 vision. We have been sent off to get electroretinograms and photos of his eyes, but because the issues caused in the eye in MPS II cannot be treated with glasses or surgery, drops or anything else, I’m going to push them off. We have enough appointments right now and the information will not do anything for Trey.

The procedure was 4 hour and 15 minutes. Trey’s surgeon, Dr. Verchere, booked him for 3 hours, thinking it would be plenty time. There was a lot of scar tissue and a lot of tenosynovium (what builds up in the carpal tunnel in people with MPS and causes compression of the nerve, and also, what Dr. Verchere cleared out of Trey’s hands 3 years ago). They also didn’t have a nerve stimulator that worked. Dr. Verchere tried 5 different nerve stimulators; people from departments all over the hospital brought their nerve stimulators, but none of them were consistent. So, she could not tell us if Trey’s nerves were damaged during the surgery or not. She says her gut feeling is that the surgery went well and his nerves should be fine, but she was frustrated to tell us she couldn’t give us an accurate answer. The answer will come over the next weeks as we watch Trey use his new hands.
Dr. Verchere commented that Trey’s nerves didn’t seem overly compressed. There was a lot of build up, but the nerves weren’t at all dented, as they can be. She says moving forward, she doesn’t know what to recommend regarding testing and potential future surgery. I’d like tests at some point to have a new baseline for where Trey is at after this procedure, but Dr. Verchere doesn’t know what she’d do with that information. She doesn’t know how much benefit there would be vs. risk of more scarring, damage to the nerve during the procedure and of general anesthetic. Not the most satisfying surgical results, but Trey’s hands are clear again, I trust Dr. Verchere, and here we are.
In some ways it gets easier in that we know what to expect, we’ve been here before, but in other ways, not so much. Yesterday especially, but the past few days have been rough. I think it’s a combination of all the appointments (orthopedics, ophthalmology, speech, occupational therapy, osteopathy, neuro, general pediatrician), monthly general anesthetics for which we need to keep Trey healthy, bimonthly surgeries (right now the surgeon is back there with my sleeping son…), being out of town for half the month, trial life, jet lag, feeling sorry for my child’s lack of ability to go swimming, for playdates, and participate in all the other things his siblings and friends get to, and for me, not fitting into life as everyone around us lives it… It’s been hard and isolating. And that’s all with intense gratitude for our opportunity within this trial.
This morning I was woken up by a phone call from Surgical Daycare asking us to come in early. The first ‘case’ of the day had been cancelled. We had decided to bring Avery and Sadie to the hospital with us because firstly, they wanted to come and be included and secondly, in all honesty, I want them to have a clue what it is that Trey goes through. So, we woke everyone up and got on our way.
Right now, we’re waiting for Trey to wake up. With the anesthetic they used to prevent nausea and vomiting, due the lengthy procedure, the drugs will have gone beyond his blood stream and into his fatty tissue, meaning he’ll be sleepy for longer because the drugs will take longer to exit his system.
In trial news, I found out last week that because we live in Canada, there will be an additional step in getting Trey’s IT infusions set up at BC Children’s Hospital. In addition to negotiating funding and ethics with the ‘home’ hospital, Shire needs to apply to Health Canada for a Clinical Trial Agreement (CTA). Because Trey will be the first child to participate in the trial in Canada, Health Canada will need to approve this trial existing on Canadian grounds, which means we will likely be traveling to UNC for a longer period of time for monthly doses. Breaking new ground takes time… the good news is that we don’t have to wait for treatment while we wait for beaurocracy and institutions do their thing. Last time we had to wait and watch Trey’s dsease progress while Health Canada and the BC government and BC Children’s hospital negotiated the cost vs. benefit of saving Trey’s life with IV Elaprase. This is definitely an easier place to breathe.
Oh, to the ebb and flow of life…

I got a call today. It was a long distance call and I thought it was a telemarketer. I was about to tell this woman I was busy, when she said she’d read my blog and she had some questions about the IT trial. She has a son with MPS II. She wanted to know what effects I am seeing in Trey as a result of the trial. I told her what I’ve observed, as well as what other parents have reported and what tests have demonstrated in the kids in the trial thus far: stabilization, no further decline, IQ’s going back up, an improvement in behaviour and hearing… Silence on the other end. Then the sound of tears. Then more silence. Then an apology. I’ve become so accustomed to talking about the trial (and MPS), that I can talk about hydrocephalus, brain decline, clawed hands, enlarged livers, hearing loss, hearing aids, and the list goes on and on and on… that I don’t always remember the effect of my words. When this woman’s son was diagnosed, she was told her son was going to die. I just told her that there’s something out there that could keep him alive. I’d cry too.
It brought me back to all the other parents I’ve spoken with upon diagnosis. I’ll never forget the first time I spoke with DK, Robb, Jamie, Sarah, Jen. They contacted me when their kids were diagnosed. You get this call from someone you don’t know, often at an unexpected time, and within seconds, not even minutes, you’re sharing your utter breath-taking devastation and deepest fears. There’s no point beating around the bush because you’ve been there. Hell. And you know what it’s like. You can’t breathe, you can’t sleep, and you don’t know how or why to go on.
I’ll also never forget the first phone calls I made. I was drowning and grasping for anything or anyone to give me some air. Simon, Marie, Kirsten, Carolyn. I have made my share of calls, sobbing, because I didn’t know how to dig myself out of this deep deep hole.
I am glad this mum called me. It takes courage. And it gave me pause. I’ve posted many many times about my gratitude for this trial, for life, for Trey and my family, but this post of gratitude is for my MPS community. For answering the phone and listening when you didn’t know who I was. For sharing your story, experience, advice, and knowing, year after year. For making me feel I’m not alone and crazy. I love you.
The above picture is DK’s son Tyler. Beautiful.

For anyone affected by a rare disease worldwide, and for all of us working towards an Orphan Drug Policy in Canada, the below thesis is an excellent source of information and I think, well worth the read. Below is the abstract, click the link for the full thesis: https://circle.ubc.ca/handle/2429/39881
“In the context of Canada’s publicly funded universal health care system, access to potentially life-saving and/or life lengthening orphan drugs costing anywhere from $100,000.00 to $850,000.00 per patient per year is a complicated matter.  This study is an anthropological examination of the debates surrounding ‘expensive drugs for rare diseases’, a term that has come to represent the costly treatments developed for rare metabolic diseases like Mucopolysaccharidosis, Pompe Disease, Fabry Disease, and Phenylketonuria.
This study was conducted in British Columbia, Canada.  It is based on several months of participant observation in hospital, industry, and patient advocacy contexts, as well as 14 semi-structured interviews conducted with the different stakeholders in the debate: patients and families, health care professionals, representatives of the provincial government’s Ministry of Health, pharmaceutical companies, and patient advocacy groups.  This study looks at discussions of authority, responsibility, and rights to health care/health technology.  It examines how complex systems of relationships shape these discussions in a particular time and place, and how the competing cultural models of publicly funded health care and profit-based pharmaceutical policy and industry operate in the context of extremely expensive drugs. The body of literature on orphan drugs in the social sciences/humanities is very underdeveloped, and there are no known comprehensive social scientific/ethnographic studies of the metaphors, constructs, and cultural context of debates surrounding orphan drugs/expensive drugs for rare diseases.  This study attempts to fill some of these gaps by looking at the complexities of different stakeholder arguments and their structural and discursive context.
In attempting to reconcile and solve the problems of accessibility to EDRD, the different stakeholders directly implicated in the debate mobilize culturally shaped notions evidence, accountability, fairness, and responsibility.  This study demonstrates that the problems, pitfalls, and provisional solutions articulated by the different people implicated in this debate throw in to relief the many contradictions between orphan drug policies, neglected diseases, drug regulation/assessment practices, and the relationship between pharmaceuticals and society.  These frameworks and competing cultural models are creating tensions that may be irreconcilable with a publicly funded health care system.”

Just before his third birthday, after six monthes of fighting with the BC government, Trey became the first person in Canada to be approved for Elaprase, a life-saving IV Enzyme Replacement Therapy. This past August Trey qualified, after years of waiting, for a life-saving IT Enzyme Replacement Therapy trial at UNC. He had his third monthly IT dose of Elaprase this morning. White count less than one. 30 more mg of enzyme floating around in Trey’s brain.
The gratitude never gets old. I have had to fight and advocate and wait for every treatment Trey’s received. To date, even when Trey’s had the flu and not been able to get his IV Elaprase doses on time (you can’t get your infusions when sick, as that increases the chance of reaction), I’ve made them up, doing infusions every five or six days until he’s back on track with his weekly treatment. Trey’s missing an enzyme. I’m not going to give up on any chance to get that enzyme into his body.
Now it’s the IT drug. After years of waiting, hearing about the possibility of an intrathecal trial, but not knowing if it would come to fruition, and then for Trey to actually be cognitively intact enough to qualify for the trial once it began, I am still blown away. Although the details of flying across the continent for 19 hours, missing connecting flights, leaving young children at home for ten days a month, Trey getting too many needles each month to count and now too many general anesthetics in his life to count, are not ‘easy’ realities to manage, they are small potatoes in the bigger picture.
Last night I lay in bed and could not sleep. Because I continue to be amazed by this chance we have. This past Saturday, Sadie saw a picture on our wall I’d just hung. It was me, holding a boy, dancing. She asked who the boy in the picture was. She said: ‘I know that’s you (pointing to me), but who’s that (pointing to the boy)?’ It was a photo of Trey and I, pictured above, taken a few monthes before Trey started IV Elaprase. His features and body have changed so much as a result of his weekly treatment, his sister doesn’t even recognize who that little boy was, before he started a therapy that would save his life.
When Trey started IV Elaprase, no one knew exactly what it would do for him. No one knew that five years later, his sister wouldn’t even recognize old pictures of him. Now we have this same chance with IT Elaprase. Gratitude doesn’t even begin to scratch the surface…

After spending a lot of time speaking with Trey’s plastic surgeon, Dr. Verchere, and a hand surgeon at the University of Minnesota, Dr. Van Heest, who is an expert in MPS, we have decided to go ahead and book Trey’s bilateral carpal tunnel release. Trey has surgery January 23, 6 days after we get back from January’s UNC trip.
Both surgeons agreed that we go ahead with another release. I spoke with Dr. Van Heest for the first time, back in June. Trey had nerve conduction and EMG tests in February 2011, two years after his first carpal tunnel release. The tests showed Trey had carpal tunnel syndrome, but Dr. Van Heest could not tell from one test, if this carpal tunnel syndrome was a result of damage done before his first surgery that was permanent and irreparable, or if it was due to new GAG deposits, since surgery (in the future we will be doing EMG’s and nerve conduction tests yearly… live and learn). So, we did another EMG and nerve conduction test last month, in November. November’s tests were considerably worse than February’s results, so Dr. Van Heest said she would go ahead with another release. This damage is not residual, it is new accumulation.
So, here we go for a second carpal tunnel release. One of my biggest questions, was how many releases can one do in a lifetime? If Trey’s carpal tunnel syndrome comes back again after this release, what do you do? Dr. Van Heest said she has never done three carpal tunnel releases before (she has also never heard of anyone else who has done it before either), only because it has not been necessary. With enzyme replacement therapy, kids are living longer and we would be entering uncharted territory.
I asked Dr. Verchere what the concerns of repeated surgeries are. Apparently, doing repeated surgery in one area is more complicated, but not impossible. Dr. Verchere said that when you do surgery a first time in any given area, it is easy to see the tissue plains. First there is skin, then fat, then muscle and bone. A scar joins all these planes together, so it is more difficult to see where you’re going. As far as my understanding goes, you have to take a good look around because not only do you have to make sure you’re in the correct plane, but scar tissue might lead you to miss something if you see scarring and don’t look farther (MPS folk and doctors, please correct me if I’m wrong, I think it’s fascinating!).
For those of you who are still interested, Trey’s sensory nerve conduction results were absent from the beginning, so Dr. Van Heest can’t tell if it’s better or worse. It is his motor nerve conduction that we can assess results from, so it is his motor function that has gotten worse in the past year (it did improve after surgery, but now is even slightly worse than before his first surgery). In addition, Trey’s right hand is worse than his left and surgery did not help his median right latency. Sorry to bore those of you who are not interested, I am providing this info for all the other MPS families out there, if they come across this uncharted land! When no one knows what to do, the more information parents or adults with MPS have to work with, the better!
Dr. Verchere also commented that Trey’s hand function (she watched him eating pistachios) is good and is much better than she would have guessed from his EMG & nerve conduction results. However, despite this fact, both surgeons said to go ahead with surgery. I’m sad for Trey that he’s getting put to sleep an average of two times per month these days, but you do what you’ve gotta do. And then you adjust.
Onto osteopathy! Trey saw Carolyne, his osteopath, again today. Last visit, after his first dose of IT enzyme, Carolyne said she could feel the enzyme in there and that his brain felt different, but that the enzyme had not fully absorbed into the tissues of the brain. This time was different. She said Trey’s brain and central nervous system was more receptive to the enzyme and it was deeper in the tissues. She said she’d like to check once more after his next IT dose, just to see one more time what the enzyme is doing in his brain, but she said all she did today was help get the CSF moving from his lower to upper spine and brain a little bit better. She also said his brain and central nervous system feels completely different from what it felt like prior to IT dosing and that she thinks the IT enzyme is doing great things for his brain. I would think and hope so, but it is great to hear!!
Now I’m going to go enjoy the holidays. 🙂
Love Deb

My eighty-six year old grandma, Omi, got her first ever computer and a wireless internet connection about a month ago, so she coud read Trey’s blog (she lives a five minute drive from us and comes over at least 5 days a week, but as she explained, with three kids, there is never time to talk). My dad emailed me last night, telling me Omi’s wondering why I haven’t blogged how the past week at UNC has gone, so this one’s for you, Omi.
Fortunately, the lack of update means that everything has gone well. Trey got his 30mg dose of Elaprase into his central nervous system by lumbar puncture Tuesday afternoon and he spent the rest of his day and night in the Short Stay Unit. Although he didn’t sleep AT ALL that night, which led to a few days of recovery, it’s peanuts in the scheme of things. Wednesday morning Trey was transferred to the CTRC (Clinical Trial and Research Centre) and there he stayed until Friday morning.
Wednesday we had a visit from Chris Dutcher, a man we met for the first time, who has MPS II. He brought treats for Trey and stayed for quite a while. We also met a local Shire rep. Between Chris and this Shire rep, I am learning a lot about health coverage and rare disease treatment in the US!
Thursday Trey got his IV Elaprase infusion. Otherwise things were uneventful. We’re making good use of the inpatient playroom, which is open three times daily, we make the long trek across the hospital to Starbucks daily, so Trey can get some ‘bubbly water’ or vanilla or chocolate milk, and otherwise, we find ways to fill the day. When the ward is quiet, we play leap frog, soccer or hide & seek in the hallways. Colouring and Nick Jr. also help. The great thing about this visit is that Trey did not have an IV. On the first, sixth and 18th visits, Trey has an IV in for 4 days and is really limited in terms of what he can do. This trip was easy in comparison.
Once discharged, while Trey and Ryan spent the weekend partaking in Five Guys and UNC sporting events, I spent the weekend with a friend and her family. Her son also has MPS II and although I’ve known her for over two years and shared many many tears, fears and questions with her, we’ve never met. It was surreal and wonderful. She is family.
Today we met with Dr. Muenzer and Heather for a brief neuro exam, which turned into a two hour conversation. Dr. Muenzer is unbelievable. I continue to be amazed by his compassion, respect, collaboration, and consideration, in addition to his knowledge and experience. The amount of time he spends with families is astounding. We don’t yet have dates for a port placement (we didn’t end up getting to meet with the neurosurgeon), but we’re hoping to link it up with his end of study tests, between dose five and six, which could mean we’ll spend all of March and half of April in NC. We head home tomorrow, which could not come soon enough to our little loved ones waiting at home. We can’t wait.
My friend, Melissa, whose son Case I’ve talked much about and is also in the IT trial, just wrote a great blog (with graphs!) of Case’s changes since beginning this new drug. It’s worth a read. Interesting and informative: http://networkedblogs.com/rL5Xj?mid=5596790
And seeing that we’re nearing the end of the year, it’s impossible not to reflect on our past year. One year ago, we didn’t know what our future held. We dreamt of saving Trey’s life and hoped that Trey would qualify for the intrathecal trial that would make our dream come true, but we had no idea if it would happen. We also held a star studded gala that raised double what any of our previous fundraisers had, so we were able to issue a request for MPS II Research Grant applications for $100,000. What an overwhelming and awesome year it’s been. To my family and friends and all the people I’ve met this year, thank you for helping make it the year that it was. I wish you all the best for 2012.
Love Deb

Our second online auction is up! It will be live from Dec. 14-23 and will benefit the MPS II Research Fund (to read more about the fund and how it works, please visit www.treypurcell.com). Click on the links below to bid on items and donate for research to a cure for MPS II Hunter Syndrome!

1. 15-MINUTE Skype Chat with Ryan Robbins & Robert Lawrenson from Sanctuary

http://www.ebay.ca/itm/270874204617?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

2. 15-MINUTE Skype Chat with Ryan Robbins & Robert Lawrenson from Sanctuary

http://www.ebay.ca/itm/270874205815?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

3. Autographed Erik Christensen Rangers Jersey Signed by the Entire 2010/2011 Team

http://www.ebay.ca/itm/270874233611?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

4. Men’s Marine Canvas Jacket worn on the Set of Stargate Atlantis (Authentic)

http://www.ebay.ca/itm/270874212306?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

5. Autographed Star Trek Poster signed by Six Characters

http://www.ebay.ca/itm/270874218580?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_898

6. Autographed Star Trek Cast Photo of Vaughn Armstrong

http://www.ebay.ca/itm/270874228121?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

7. Autographed Stargate Universe Cast Photo of Mark Burgess

http://www.ebay.ca/itm/270874229620?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024

8. “Cher Rinoceros” by Graciela Rodo Boulanger

http://www.ebay.ca/itm/270874236363?ssPageName=STRK:MESELX:IT&_trksid=p3984.m1555.l2649#ht_500wt_1024
Our last online auction, which was live in September, raised over $20,000 for MPS II research. Our Once Upon a Cure Gala on September 24, 2011, raised enough to fund a $100,000 MPS II research grant. Click here to read about the request for applications we currently have out (the application deadline is February 29, 2012) and to read about the grants we have funded in the past.
Thank you in advance for your support. As I write this, I am sitting in a hospital in North Carolina while Trey participates in a clinical trial for a treatment that will hopefully extend his life. However, only a cure will save it. You are making that happen.
 

Trey’s second IT Elaprase dose, of 30mg, is complete. He received it this afternoon via lumbar puncture under general anesthetic. Long story as short as possible, a doctor accesses Trey’s central nervous system (either by lumbar puncture or by intrathecal device) and draw off CSF (cerebrospinal fluid). They send the fluid to the lab and run a number of tests, but the only one that matters for dosing, is the white count. Trey’s white count was under one, which is great. Under 5 is normal, over 30 and you don’t get your dose, over 100 and you’re out of the trial (it’s how they determine if a device is failing or if a child may be having a reaction). So, one the lab results came back, Trey got his dose. Everything went well.
There is so much going on with this trial, things are changing daily. Even being down here 2 monthes ago, which seems relatively recent, I still had two pages of questions for Dr. Muenzer. And then, after being here for two days, all sorts of new information comes up. About the device: new breakages, new fluid accumulation, new leakage, new removals, a potential new device. About the drug: potential reactions, some IQ’s increasing dozens of points after receiving intrathecal drug, some stabilizing, hydrocephalus being resolved… the list goes on and on. I have had hours and hours and hours of conversation over the past two days with parents of kids in the trial, doctors and nurses involved with the trial. My mind is in a haze. High. Low. Fear. Elation. But above all, gratitude and appreciation. To have this chance. Enzyme is floating around in Trey’s brain. Indescribable.
Right now, Trey is in hospital and will be until Friday. Before we come back home we will see the neurosurgeon and talk about new device placement. We will also get projected dates for dosing over the next six monthes, end of study testing dates (the study is only 6 monthes long, and because Trey missed his second dose in November, he will have his end of study tests after 6 monthes of the trial, but only 5 doses. However, after the six monthes, Trey does not stop drug, he will roll right into the extension study), and probably, new port placement dates.
In addition, we have an appointment with Trey’s hand surgeon on December 21 to discuss a bilateral carpal tunnel release, so we will be preparing for that in the new year as well. I am sure glad I don’t have anything else on my plate because this right now is a full time job!
Thank you everyone for all that you are doing. To our babysitters in North Vancouver, to Jamie for making us feel at home in NC, to Christopher for visiting, to Liz for creating the beautiful header for this website, to all our family and friends who have remembered, hugged us, given us your positive thoughts. You have made it a less lonely road.
Love love love,
Deb

Shire honors 14 caregivers through inaugural international recognition program
DUBLIN, Nov. 18, 2011 /PRNewswire/ — Fourteen caregivers from around the world have been announced today as Recipients or Distinguished Honorees in the inaugural Shire BRAVE Awards, an international program that aims to recognize everyday people who serve as caregivers. Launched by Shire in June 2011, the BRAVE Awards is the first and only awards initiative of its kind that honors the courage and dedication of non-professional caregivers, the unsung everyday heroes who provide regular and consistent care for other people.
“Helping another person through consistent, dedicated care is one of the most generous acts and we believe that such caregivers deserve to be recognized and celebrated,” said Angus Russell, Shire Chief Executive Officer. “This unique program strikes to the core of Shire’s purpose of enabling people with life-altering conditions to lead better lives. We are privileged to honor these individuals who are models of courage and selflessness through their devotion to caring for others, often in the face of adversity.”
Shire received nearly 400 nominations from seven countries over 10 weeks for the BRAVE Awards. The BRAVE Awards Selection Committee, which included select Shire employees and Gary Barg, Editor in Chief of Today’s Caregiver andcaregiver.com as well as author of The Fearless Caregiver, reviewed and ranked the nominations based on the qualities that define Shire’s own BRAVE culture: respect, courage, dedication, impact and patience. Shire’s nearly 5,000 employees from across the world helped to select the Recipients and Distinguished Honorees from the pool of eligible finalists.
Shire is proud to honor 14 people as part of the inaugural BRAVE Awards; 10 Recipients each will receive $10,000 USD or the local currency equivalent, and four Distinguished Honorees will each be awarded $2,500 USD or the local currency equivalent, for a total of $110,000 USD.
“The BRAVE Awards is an outstanding program that recognizes those who selflessly dedicate their lives to the health and well-being of others who need it most,” said Mr. Barg. “The 14 inaugural recipients and honorees are heroes who are truly deserving of this recognition.”
2011 BRAVE Awards Recipients*
While they come from a range of different backgrounds and face unique challenges, the Recipients and Distinguished Honorees are connected by their tireless dedication and commitment to others.  To read more about and hear from each Recipient and Distinguished Honoree, visit www.ShireBRAVEAwards.com
RECIPIENTS:
Zarrar Beg: Zarrar cares for four handicapped children, including his three daughters, who have a form of Mucopolysaccharidosis (MPS), and his autistic son, all the while striving to fill every day of their lives with as much joy as they’ve given him and his wife.
Natalie Gibbs: For 14 years, Natalie has selflessly acted as her daughters’ “arms and legs” after they were diagnosed with Spinal Muscular Atrophy (SMA), a progressive disease that leads to loss of muscle control and movement.
Joan Metz: Joan has spent nearly 12 years caring for her adopted daughter Sophie, who suffers from the effects of Shaken Baby Syndrome, making sure that she receives speech therapy and gets the regular care she needs to thrive in a loving family environment.
Christine Muller: Thanks to Chris’s strength, her son Ben, who has Duchenne Muscular Dystrophy, learned to ride a bike at six years old, walked until he was 14 years old and is now a thriving 26 year-old.
Ronna Reid: Ronna has been her daughter Amanda’s primary caregiver following her Rett Syndrome diagnosis in 1991, all while raising three other children diagnosed with other various disorders and advocating tirelessly for Amanda’s future.
Micky Todd: For over a decade, Micky has dedicated his life to supporting whoever in his family is in need of help, beginning with his father-in-law, and including his own father and later his mother, who is dealing with both Alzheimer’s and arthritis.
Rosemarie Veliz: Rosemarie has committed herself to caring for her son, Chase, who was diagnosed with a form of Mucopolysaccharidosis (MPS), and to becoming an integral part of his medical team over the years.
Jennifer Wheeler: Jennifer has spent the last nine years dedicated to her son, Brandon, who was involved in a motorcycle accident that left him paralyzed from the neck down.
Matthew Wolfson: Matthew stayed by his now fiancée’s side when she was in a coma following an accident; after she emerged from the coma, Matthew stepped in to support her in their life together.
*One Recipient requested that her information not be shared in this press release.
DISTINGUISHED HONOREES:
Jennifer Alawadi: After marrying her husband, who suffered from Cystic Fibrosis, Jennifer focused on fighting for his quality of life – including a successful legal battle to win insurance coverage for his lung transplant surgery.
Douglas R. Cook: Doug has committed himself to providing the best life possible to his wife, Radene, who suffered a concussion and severely damaged spine after a plane crash.
Maria Douglas: Maria dedicated herself to her husband’s recovery after he was injured as a soldier in Iraq, giving up her job twice to become his full-time caregiver.
Vickie Whalen: Vickie has spent more than nine years taking care of her son Brandon, who was born with Agenesis of the Corpus Callosum (ACC), a disorder in which the structure of the brain that connects the two hemispheres is absent, and helping him live a joyful life.
The Shire BRAVE Awards are presented in the spirit of Shire’s BRAVE culture, which fosters an environment that encourages its employees and partners to act with integrity; create innovative solutions that make a difference; act on the needs of others; strive to be better every day; and to exemplify trust and openness through teamwork. Nominations for the 2012 Shire BRAVE Awards are expected to be open on or about March 15, 2012.
Notes to editors
About the Shire BRAVE Awards
The Shire BRAVE Awards is an international program created by Shire to honor the courage and dedication of individual caregivers. For the Shire Brave Awards, a caregiver is defined as an individual who is providing regular, consistent care for another person(s). The caregiver cannot be a professional healthcare provider and cannot be compensated in any formal way through their efforts as a caregiver.  The caregiver can be a relative, neighbor, or friend who gives their time, support, and compassion regularly, helping another person(s) who is (partly or wholly) unable to care for themselves due to health issues, injury, or other extenuating circumstance. For further information on the Shire BRAVE Awards, including official rules, legal disclosures, and privacy policy please visit www.ShireBRAVEAwards.com.
SHIRE PLC
Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder, human genetic therapies, gastrointestinal diseases and regenerative medicine as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.
For further information on Shire, please visit the Company’s website: www.shire.com.