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Biogen Advances Once-Yearly SMA Therapy Salanersen to Phase 3 After Positive Phase 1 Results

Biogen Inc. (NASDAQ:BIIB) is one of the most undervalued US stocks according to analysts. On June 25, Biogen announced positive topline results from an interim analysis of its Phase 1 study for ...
EurekAlert!

Researchers evaluate the benefit of dual therapy for children at risk for spinal muscular atrophy

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...
equities

Genentech’s Spinal Muscular Atrophy Therapy Shows Significant Improvement in Motor Function

Spinal muscular atrophy (SMA) is a progressive genetic disease that causes devastating muscle atrophy and disease-related complications. SMA is the leading genetic cause of death in infants and ...
Healio

Spinal muscular atrophy therapy leads to sustained motor function improvement

Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...
The American Journal of Managed Care

Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA

Apitegromab met the primary endpoint in the SAPPHIRE trial, showing significant motor function improvement in SMA patients compared to placebo. Scholar Rock plans to submit a biologics license ...
GEN

Base Editing Strategy for Spinal Muscular Atrophy Rescues Patient Fibroblasts, Reverses Mouse Pathology

A new study describes a base editing method for treating spinal muscular atrophy (SMA). This supports early signs that “single-shot” treatments from clinical trials using genome editing technologies ...
Medscape

Gene Therapy Improves Movement in Older Children With SMA

A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...
Rutland Herald

UAE’s Medcare Treats First International SMA Patient With Revolutionary Intrathecal Gene Therapy

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a three-year-old patient from Turkey. This milestone ...
EurekAlert!

Promising results from first prenatal therapy for spinal muscular atrophy

(MEMPHIS, Tenn. – February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children’s Research Hospital led the ...
Medscape

Gene Therapy Tops Nusinersen in Spinal Muscular Atrophy

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

'I want to tell Jesy Nelson, there's hope', says mum of child with SMA

A County Armagh mother, whose five-year-old daughter has Spinal Muscular Atrophy (SMA), has said singer Jesy Nelson should know "there is light at the end of the tunnel". The former Little Mix star ...