Doug Penner will hopefully be feeling a little more energetic by the time Sunday鈥檚 motorcycle ride in his honour kicks off.
The 14-year-old was diagnosed with Duchenne muscular dystrophy (DMD) when he was two. It鈥檚 a commonly fatal genetic disorder that affects approximately one in every 3,500 to 5,000 male children.
For the past 11 years now, Doug鈥檚 father Cam 鈥� and his entire family 鈥� have hosted the Ride for Doug each June in 91原创.
This ride has raised more than $175,000 for Muscular Dystrophy Canada and been a constant morale booster for Doug.
Meanwhile, Doug has continued battling his disorder, with a number of ups and downs 鈥� through the years.
The past year has been particularly hard on him, explained his father, Cam.
Doug was part of a drug trial for Drisapersen since 2011, but last year that medication was rejected by the Food and Drug Administration. Consequently, when the study was discontinued and the medication was no longer available, Doug鈥檚 health began to deteriorate, Cam said.
鈥淔or the past yar, we have watched Doug begin a decline in strength and endurance. His muscles are weakening, despite receiving the best care possible. The one medication that seemed to make a difference sitting on a shelf, slowly expiring,鈥� explained Dad.
But as of Tuesday, the situation has changed. While the drug has still not been approved, and is not expected to be, whatever quantity was left from the study has been transferred from the pharmaceutical company to an organization called CureDechenne.
鈥淒espite everyone involved really wanting to make this abandoned drug available, the paperwork and approvals process was formidable,鈥� Cam said. 鈥淭hanks to a small but very dedicated Duchenne community we received word that the drug was going to be made available again.鈥�
Now, after continued lobbying for access, those leftover drugs are being made accessible to just a few of the past recipients. And Doug is among them.
鈥淏ut not every boy with DMD can access this. There are probably only five boys in Canada who are eligible to participate,鈥� Cam added.
Doug received his first treatment earlier this week.
鈥淲hat does this mean to us?鈥� Cam said. 鈥淭here are so many different emotions. Relief that the long waiting and uncertainty have ended. The frustration that Doug lost an entire year鈥檚 worth of mobility while waiting. And, lots of hope that we will once again see Doug stabilized like he did all those years on the medication.鈥�
Based on their experience the last time the drug was halted for a year (a few years back), the Penners expect that during the next three to four months Doug鈥檚 declining strength will stabilize.
鈥淲e anticipate him having longer endurance and better balance. To feel more energetic. We don鈥檛 expect to see a lot of strength increases 鈥� that doesn鈥檛 seem to be how this drug works 鈥� but with DMD a significant slowing of decline is a major victory,鈥� Dad said.
Practically speaking, it means the Penners will be taking Doug into BC Children鈥檚 Hospital once a week for treatment, where he鈥檒l be hooked up to an IV for several hours as the infusion takes place.
That鈥檚 a lot of missed school, Cam admitted. But it seems a small price to pay.
鈥淚 don鈥檛 know exactly how much of the drug is left,鈥� he said, noting a year鈥檚 worth has already been transferred to BCCH for Doug. Cam hopes there鈥檚 more to follow.
鈥淗opefully, researchers can make more progress on finding a more permanent cure,鈥� he said, admitting he鈥檚 burned out emotionally from this fight but remains constantly optimistic.
鈥淣ew technologies are always on the cusp of something great. This special access program buys us some of that time.鈥�
In the meantime, he鈥檚 now gearing up for this Sunday鈥檚 (June 4) ride that is expected to see about a hundred motorcycle enthusiasts leave the South 91原创 Church (20098 22nd Ave.) at exactly 1 p.m. and take a three to four-hour ride through the rural areas of the Fraser Valley before returning to the 91原创 church for a barbecue.
For more information or registration, visit: .
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