An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

Doctors diagnosed Carmen Lopez with spinal muscle atrophy (SMA) at birth, a rare genetic disorder that affects motor neurons ...

Expert diagnostic services and treatment are provided for patients with common and rare disorders of the muscular system, including muscular dystrophies. Myopathic (myo = muscle, pathy = disease) ...

Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS ... a dozen medicines for neurological disorders, including multiple ...

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...

Berlin, Germany, and Research Triangle Park, N.C., USA, Feb. 19, 2025 (GLOBE NEWSWIRE) -- Not intended for UK MediaRegenerative Medicine ...

Roche has announced that a tablet formulation of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) has been ...