Bipartisan Congressional Action Advances Critical Programs for Rare Neuromuscular Disease Research and Care Muscular ...
Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.
Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...
A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...
A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...
Add Yahoo as a preferred source to see more of our stories on Google. Amy is campaigning for early screening after her son Oakley was diagnosed with spinal muscular atrophy (SMA) [Family handout] The ...
The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...
Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...
"The strongest, most resilient babies," the Little Mix singer said of her daughters Ocean Jade and Story Monroe ...
Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced interim data from a phase 1b study of ABX1100 ...
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