Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately ...

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

EAST HANOVER, N.J., Nov. 24, 2025 /PRNewswire/ -- Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma ® (onasemnogene abeparvovec-brve) for the treatment of ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

The Integrated Health Clinic at University Medical Center New Orleans with LSU Health New Orleans is one of only six teams ...

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...

For decades, high cholesterol has meant a lifetime of daily pills, repeat blood tests and constant reminders that the biggest ...

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...