Objective The aim of this study was to assess early language acquisitions in treated individuals with spinal muscular atrophy (SMA) type 1 and in infants identified by newborn screening (NBS). Methods ...

Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, interim results show.

Amyotrophic lateral sclerosis (ALS) is a lethal neurodegenerative disease prominently featuring motor neuron (MN) loss and paralysis. A recent study using whole-cell patch clamp recording of MNs in ...

In perhaps a first in the country, a newborn found carrying the SMN1 gene mutation for Spinal Muscular Atrophy (SMA) through prenatal genetic screening is being administered presymptomatic ...

Thiruvananthapuram: For the first time in India, pre-symptomatic treatment to an infant, diagnosed with spinal muscular atrophy (SMA) within days of b.

Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments ...

5q-Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disease with an incidence of 1 in 14,848 births.1 SMA is characterised by proximal muscle weakness and in severe ...

Sarepta Therapeutics (NASDAQ: SRPT) fell ~38% in the premarket on Monday after the company reported a second fatality from acute liver failure in a patient treated with Elevidys, a gene therapy ...

EP: 3. Key Takeaways From DMD Treatment Data EP: 4. Overview of Spinal Muscular Atrophy EP: 5. Gaps in Care for Childhood SMA EP: 6. The Impact of Intrathecal Onasemnogene Abeparvovec Therapy in SMA ...

The prognosis of spinal muscular atrophy (SMA) depends on many factors, including disease type and severity, age at symptom onset, SMN2 gene copy number, genetic modifiers, and the therapeutic support ...

RTX and Northrop Grumman conduct successful rocket motor tests for U.S. Army's Next-Generation Short-Range Interceptor Jun. 05, 2025 9:00 AM ET Northrop Grumman Corporation (NOC), RTX ...