Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

"The benefits of newborn screening identified in the study presented here encompass not only the achievement of motor milestones, but also a reduced need for ventilator support or tube feeding and a ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and ...

"Our study demonstrated that profound impairments in swallowing are extremely common within the first year of life. This of course has tremendous clinical relevance as it highlights the importance of ...

Articles by ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017 259 Prospect Plains Rd, Bldg H Cranbury, NJ 08512 ...

Please provide your email address to receive an email when new articles are posted on . Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

INDIANANAPOLIS, Indiana -- A mother in Indiana refused to give up on her infant son suffering from a neuromuscular disorder, even when an insurance company and a $2 million price tag stood in her way.

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs. Previously, ...