A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here are ...

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...

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 ...

The former Little Mix singer is on a career break as she focuses on her 10-month-old twins Ocean and Story and to campaign for testing at birth for SMA1 (Spinal Muscular Atrophy Type 1), a severe form ...

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

Famous American singer Drake Bell, known for his roles in shows like Nickelodeon’s Drake & Josh, visited a very special baby in Ensenada. He is Rafael, also known as Rafita, a 1-year-and-8-month-old ...

Children born with a rare neuromuscular disorder are being given another chance in life thanks to a new treatment experts are ...