Rare diseases affect millions, but most remain undiagnosed and untreated. Learn about Rare Disease Day 2025, patient ...

BATON ROUGE, La. (WAFB) - In the late days of February, we are continuing to spread awareness during American Heart Month. The American Heart Association says heart disease is the leading cause of ...

There are several treatment options available for spinal muscular atrophy (SMA), a rare genetic condition characterized by progressive muscle weakness and wasting. The disease mainly affects motor ...

cThe Dubowitz Neuromuscular Unit, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre and Great Ormond Street Hospital NHS Foundation Trust, ...

The intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease ... Early outcomes in three human volunteers showed ...

31 said his staff could not use official resources, including their time, to host events related to cultural awareness months, including Black History Month, Women’s History Month and National ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative ... Syrian Associated Charities (ALSAC), the fundraising and awareness organization of St. Jude.

What is Evrysdi for SMA? Evrysdi (risdiplam) is a disease-modifying therapy, taken daily, that’s approved for children and adults with spinal muscular atrophy (SMA). It is the only approved SMA ...

The disease Mitchell was diagnosed with, back in April 2017, was spinal muscular atrophy (SMA)—a rare ... fundraising and spreading awareness about SMA. His Instagram account, SMA Warrior ...

The child was conceived with a genetic condition known as spinal muscular atrophy, which affects motor neurons that control movement, and leads to progressive muscle weakening. About one in every ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...