DUBAI, United Arab Emirates--(BUSINESS WIRE)--Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA). “Our results underscore the heterogeneity of oral function effort across ...

Biogen BIIB recently announced that the FDA has granted Breakthrough Therapy designation to Salanersen, an investigational ...

Add Yahoo as a preferred source to see more of our stories on Google. May 1—At the age of 23, Chloe Caldwell is learning to drive. She had her first lesson in mid-April, but won't be able to get back ...

The risk of scoliosis was largely related to what type of SMA a patient had, with those with type III having the lowest risk and those with type II developing scoliosis at a later age than those with ...

Dubai’s Medcare Hospital Becomes the World’s First to Treat an Adult Spinal Muscular Atrophy (SMA) Patient Outside the US Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world’s first ...

Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Nearly six years ago, the Food and Drug ...

As treatment for spinal muscular atrophy (SMA) has drastically improved over the last decade, so too have survival rates and the number of people with SMA living well into adulthood. In line with that ...