Biogen’s successor to Spinraza (nusinersen) has shown benefit in spinal muscular atrophy (SMA) patients who had suboptimal clinical status despite prior administration of gene therapy. In the Phase Ib ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

Union Minister H D Kumaraswamy has assured assistance for a two-year-old child suffering from a rare genetic disorder after ...

SMA mutations disrupt spinal cord development, with fewer nerve cells in early stages. SMN protein deficiency leads to a mesodermal fate bias, with fewer motor neurons. Increasing SOX2 pathway ...

Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage global genetic medicines company, announced today that the first patient has been dosed in the Phase 1/2 CHARISMA clinical trial of GB221, an ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

SMA newborn screening may help infants start treatment sooner, which researchers say could support better motor development outcomes.

US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel ...

Bangkok Hospital Introduces Precision Medicine Hub in Southeast Asia featuring Gene Therapy for Children with Spinal Muscular Atrophy (SMA) & Anti-Amyloid Therapy for Alzheimer's Disease ...

Scientists at the Tomsk Scientific Research Institute of Medical Genetics have patented a method for diagnosing spinal muscular atrophy using digital PCR. The development is designed to close the ...