Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

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 ...

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, ...

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 ...

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 ...

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...

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 ...

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 ...