Help Derek SMAshSMA!

Story

Thank you for joining #TeamDerek! as Derek and his family work to SMAshSMA!, both for Derek and for every other family dealing with this disorder.   Derek was born on June 6, 2015, a healthy 8 lbs, 11 oz and 22 inches long.  As his parents Dave & Megan Cardenas, and big brother Alex (4 1/2) and the rest of the famil eagerly welcomed Derek, we knew that Derek had a 25% chance of having Spinal Muscular Atrophy (SMA), a form of muscular dystrophy.  Late in Megan's pregnancy, Megan & Dave learned that they are each genetic carriers of the disorder.   18 days after his birth, the blood tests came back - Derek has SMA Type 1b (onset with symptoms before 6 months old,  to date incurable and until recently with little hope.)    People with SMA lack the survival motor neuron (SMN) enzyme needed to communicate between the brain and along the spine to ones muscles. For those with SMA1, the muscles slowly but surely stop working leading to death. SMA is the number one cause for genetic-based infant mortality.   Less than 48 hours  after diagnosis, Megan and Da6/26ve had identified support programs, and a clincial trial for a very promising clinical trial, and were seen  by  members of Columbia University Medical Center's Pediatric Neurology Clinical Trial team.  Derek was accepted in the Endear Clinical Trial, testing a promsing new drug that is showing stunning preliminary results.   Derek received his first treatment yesterday, July 6 - though we don't know if received the drug (2/3 do) or the placebo (1/3 participants), we are excited  about the prosects and thrilled that Derek is being cared for by the memers of the SMA Clinical Research Center team.  Even with the hope of the new drug, whether Derek is receiving it now (we won't know until the year-long trial is over) or upon approval, Derek is already showing symptoms of SMA, having lost strength in his neck muscles since birth as well as d iminished arm and leg mobility (a/k/a floppy limbs.)   Body trunk development is affected by SMA and breathing and swallowing issues are common.    There is some evidence that the trial drug may reverse some or all of this deterioration - in the meantime, Derek will need some specialized equipment for support - equipment that is very expensive and likely not covered by insurance.   Hence this campaign.      SMA infants need special carseats that allow them to recline or lay flat and strollers that do the same.   Equipment such as the Jane Matrix Light 2 car seat, https://www.youtube.com/watch?v=DYRps117ECc, and all the associated attachments will allow Megan, Dave and Alex to take Derek out safely.   Home equipment including specialized wheelchairs and standers will help Derek develop and strengthen as he grows.     And although insurance will cover some of the in-home and physical threapy care required, additional, and significant, out-of-pocket charges are a virtual certainty.  What matters most, of course, is your thoughts, prayers and and postive support, which is overflowing and  deeply  appreciated.  Offers of childcare for Alex, meals, and other assistance are already helping so much.  And for those who have asked, yes, financial support will help.   Please know that your support will help not only Derek and his family, but others as well.   As soon as Derek has outgrown or no longer needs a piece of equipment, that item will be donated to another SMA family through CureSMA.org or another well-regarded SMA support program.    Megan and Dave know how blessed they are by the support of their large circle of family and friends; they want to share that support, once Derek is done with any piece of equipment, with those less fortunate.   Please go to CureSMA.org to find out further information about the disorder and trial. Keep a look out on your Facebook feeds for some #SMAshSMA videos coming to you soon! From Derek & his entire family   - thank you so much!