For example, 3D SKMOs derived from patients with DMD have modelled TGFβ-driven fibrosis and muscle contraction failure, providing a platform for optimising exon-skipping therapies. 66 Furthermore, ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

New data from Biogen reveals higher Spinraza doses may enhance motor function in SMA patients. Read more here.

"Spinal Muscular Atrophy Pipeline"Spinal Muscular Atrophy companies are Amniotics, Biohaven Pharmaceuticals, Hoffmann-La Roche, Amylon Therapeutics, Voyager Therapeutics, Catalyst Pharmaceuticals ...

Spinal muscular atrophy (SMA) is a severe motor neuron disease caused by mutations in the SMN1 gene, leading to reduced SMN protein levels. Although postnatal treatments like the antisense ...

5K at Xavier University raise awareness for those living with spinal muscular atrophy. by WKRC. Sun, June 1st 2025 at 9:21 PM. Updated Mon, June 2nd 2025 at 11:16 PM. 4. VIEW ALL PHOTOS.

Roche’s Evrysdi (risdiplam) tablets have been approved by the European Commission (EC) to treat spinal muscular atrophy (SMA). The new, room-temperature stable formulation of Evrysdi may offer ...

BiogenBIIB said Wednesday its experimental treatment helped children with a devastating muscle-wasting disease even after they received an approved gene therapy from NovartisNVS.The company already ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA Evrysdi offers the same efficacy and safety demonstrated in ...

European Commission approves Roche’s Evrysdi tablet to treat spinal muscular atrophy: Basel Thursday, June 5, 2025, 09:00 Hrs [IST] Roche announced that the European Commission (EC) has approved a ...