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 ...

A Litchfield woman is running the Boston Marathon to raise awareness for spinal muscular atrophy and honor her daughter, who defied medical expectations by living beyond her second birthday.

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 ...

Campaigners hope pilot will result in heel prick test for rare condition being approved across Britain ...

Singer Jesy Nelson has been campaigning for better testing of SMA Type 1 follow her twins' diagnosis, but what is the ...

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 ...

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.

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 ...