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 ...

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 ...

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...

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.

Former Little Mix star Jesy Nelson has been keeping her fans up-to-date with how her twin baby girls, Story Monroe and Ocean Jade, are getting on, after their spinal muscular atrophy Type 1 diagnosis ...

Jesy Nelson is opening up about her babies’ health. After the Little Mix alum’s mom noticed that her 8-month-old twin daughters Ocean and Story—whom she shares with fiancé Zion Foster—were showing ...

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 ...