News

Taldefgrobep alfa scores orphan drug designation in Europe

The European Commission has granted orphan drug status to taldefgrobep alfa as a potential treatment for spinal muscular atrophy (SMA). The designation is given to therapies that may improve care for rare diseases. It confers certain incentives, including a reduction in some regulatory fees and a guarantee of 10…

Awareness Month shines brighter spotlight on SMA community

“Walk-n-Rolls,” storytelling, and candle-lightings mark some of the events happening this August for SMA Awareness Month, in an effort to raise disease knowledge, patient advocacy, and research funding to help find a cure for the rare genetic disease. Each August, SMA Awareness Month strives to raise awareness of spinal…

‘We are lucky and unlucky,’ SMA caregivers say of new treatments

For caregivers of children with spinal muscular atrophy (SMA), the availability of new disease-modifying treatments has offered hope — but difficulties in accessing these therapies and uncertainty about their long-term effectiveness also have fostered frustration and anxiety. That’s according to a new qualitative study, done in Canada, that aimed…

CHMP favors extending Evrysdi to newborns with SMA in the EU

The Committee for Medicinal Products for Human Use (CHMP) favors European Union (EU) approval of Evrysdi (risdiplam) being extended to cover babies with spinal muscular atrophy (SMA) younger than 2 months of age. The recommendation now will be reviewed by the European Commission, which is expected to issue…