Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

As with any chronic disease, but especially with one that limits the patient’s mobility or ability to breathe, a heavy burden falls on the caregiver, be it a healthcare professional, close friend, or ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Biogen Inc. (NASDAQ:BIIB) on Wednesday announced in a press release the topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for ...

Waking up, hopping out of the bed, and stumbling to the kitchen for a cup of coffee: It’s an everyday routine most people don’t think twice about. But for children with spinal muscular atrophy, simply ...

AKRON, Ohio (WJW) – At 14 months old, Marshall Bowling of Millersburg seems a happy little boy, but his journey to this point has not been easy. His challenges started soon after he was born when ...

In Trumbull County, a local nonprofit will once again be hiding colorful candy-filled eggs in yards this Easter to help a ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...

JESY Nelson burst into tears after a painful reminder of the struggle her twins are facing. The former Little Mix star was overcome with emotion as she opened up about the ongoing challenges, ...