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 ...

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 ...

FORMER Little Mix star Jesy Nelson has spoken candidly about the ‘heartbreaking’ health challenges her twin daughters have ...

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 ...

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 ...

(RTTNews) - Genentech, a subsidiary of Roche Holding AG (RHHBY), Friday announced a new 5-year data confirming benefit of Evrysdi for the children with Type 1 spinal muscular atrophy or SMA as they ...

What is SMA? The rare genetic condition that Jesy Nelson’s twins have - Former Little Mix star revealed her twin daughters had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1 ...