We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...

Part B was comprised of a pivotal cohort in treatment-naïve patients with infantile-onset SMA (n=75), and a supportive ... children and adults with spinal muscular atrophy (SMA). As a foundation of ...

Truist Financial analyst Joon Lee maintained a Buy rating on Catalyst Pharma (CPRX – Research Report) today. The company’s shares closed ...

Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle disorder that leaves patients too weak to walk, talk and swallow.

Spinal muscular atrophy (SMA) is a devastating ... SMA, though a rare disease, is the leading genetic cause of infant death. The severity of the condition varies across a spectrum of types.

Objectives: The aim of this study was to assess the perception of quality of life of patients with spinal muscular atrophy (SMA) and investigate whether there is a correlation between patients’ ...

Spinal muscular atrophy (SMA) is a progressive genetic neuromuscular condition ... Neurologically, the patient scores 7 points on the Hammersmith Infant Neurological Examination scale and 3 points on ...

“If you are developing a drug, for example, for infantile epilepsy ... This has recently led to an approved therapy for spinal muscular atrophy. Such technologies have immense potential to ...

Its gene therapy drugs used for treating Duchenne muscular dystrophy, infant spinal muscular atrophy, SMA-PME (spinal muscular atrophy with progressive myoclonic epilepsy), Farber’s disease, Leber ...

The 18-month-old, suffering from a rare genetic disease called Spinal Muscular Atrophy (SMA ... astonished one day to see his father wash his infant grandson's cloth diaper.

The Amsterdam-based biotech firm said that its melt curve analysis-based test is used as a first-tier genetic screening tool to aid the diagnosis of SMA.

One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder affecting infants and young children, impacting their movement and muscle function. SMA is a genetic disorder of movement ...