This is a miracle 💓 Boy with SMA Type 2 is running, standing and sitting, lifting alone. This is only possible with early treatment from birth with Spinraza. This is the new face from spinal muscular atrophy (SMA) 💪 #AdgangTilSpinrazaForAlle #AccessToSpinraza
Typical brothers playing. But can you believe one of them has SMA? Gabriel Peters was given the first FDA approved SMA treatment, Spinraza, while it was still in clinical trial three years ago. And this, friends, is a miracle. He is a miracle. The Peters family know this firsthand as their older daughter has SMA Type 2. Typically, the severity (or the type) is similar between siblings. But, with a treatment from birth, doctors now say Gabriel genetically has SMA but is asymptomatic — no type at all. Running, standing, lifting and throwing toys… all things not usually possible with this disease. This is SMA. The new SMA. And it is astounding! NEVER GIVE UP. www.NeverGiveUp.org#mynevergiveup #thisissma #miraclesdohappen #withalotofwork
ELK GROVE VILLAGE, Ill. and SOUTH SAN FRANCISCO, Calif., May 17, 2018 (GLOBE NEWSWIRE) — Cure SMA and Cytokineticstoday announced an expanded partnership to increase education, awareness and fundraising for spinal muscular atrophy (SMA). As a National Platinum Partner for 2018, Cytokineticswill lend support to several of Cure SMA’s initiatives at both the local and national level to advance understanding of, and research toward potential treatments for SMA, a genetic disease that robs people of physical strength and takes away their ability to walk, eat or breathe.
“We have enjoyed a longstanding relationship with Cure SMA and are pleased to expand our involvements, particularly at a time when more attention and research are focused on the SMA community,” said Robert I. Blum, President and Chief Executive Officer of Cytokinetics. “We remain committed to advancing therapies for adolescents and adults with types II, III and IV SMA, a population in need of new therapies to address impaired muscle function and weakness despite the promise of gene-directed therapies.”
In his own country (Denmark), he was denied access to the only treatment for spinal muscular atrophy.
Now having the parents a positive message. Her son Magnus 17 years old is being treated with Spinraza in Belgium. He has now received the 2 injection via lumbar puncture. He feels already the first improvements into the legs.
With Spinraza can treated adults as well. Countries like Germany have for all SMA types approved without age limit.
Latest study from Germany show. Spinraza works in children and adults
On Thursday, April 26, Cure SMA traveled with more than 70 spinal muscular atrophy advocates to Washington D.C. to meet with over 60 congressional offices. The advocates represented 24 states and D.C. The purpose of the advocacy day was to advance life-changing opportunities for our community through SMA research and newborn screening.