Ridhay’s parents received the devastating diagnosis just months ago, and they’ve since been living each day with a renewed ...

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy ... production of the survival of the motor neuron (SMN) protein.

Motor improvements in adults with spinal muscular atrophy (SMA ... preserved functionally connected motor neurons without increasing overall motor neuron survival.” Spinraza also restored the nerve ...

Spinal muscular atrophy (SMA) is a progressive motor neuron disorder. While there is a spectrum of ... This has resulted in major motor and survival benefits.5–7 In the past 7 years, disease-modifying ...

Digital PCR to simultaneously detect SMN1 gene defects and the number of SMN2 copies to diagnose SMA matches standard methods ...

Other lower motor neuron diseases that may cause fasciculations include post-polio syndrome, spinal muscular atrophy (SMA), and progressive muscular atrophy. Besides neurological diseases, ...

(0:54) How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit without impeding growth? Dr. Ashley E. Webb of the University of Texas ...

Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

Jokes by comedian Samay Raina - that allegedly mock visually impaired and a baby suffering from spinal muscular atrophy, and requiring a Rs 16 lakh injection to survive - have been flagged by the ...