We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio Topline results from a phase 3 clinical ...

Spinal muscular atrophy (SMA) is a rare genetic condition that impairs a person’s ability to control their muscle movement. Most types of SMA are diagnosed in infants, but the condition sometimes ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

man MS wheelchair Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the ...

In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months. Treatment with risdiplam was associated with ...

Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained and improved motor function at 24 months in children and adults with type 2 or type 3 spinal muscular atrophy, ...

Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a ...

- Motor function improvements were observed for all three SRK-015 treatment cohorts in the primary efficacy endpoints (Hammersmith scale scores) at six-months; top-line data from the 12-month ...

Early diagnosis and treatment of SMA can limit disease progression in children and adults, extending life expectancy and improving QOL. Spinal muscular atrophy (SMA) is a severe genetic condition that ...

A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children. Basel, Switzerland-based drugmaker Novartis said Saturday that interim data ...