spinal muscular atrophy

May 19, 2025

The approval of three spinal muscular atrophy therapies—SPINRAZA (Ionis/Biogen), EVRYSDI (Roche), and the groundbreaking gene therapy ZOLGENSMA (Novartis) has significantly advanced treatment options for a condition that previously had limited solutions. These spinal muscular atrophy (SMA) therapies offer patients ...

Sep 10, 2024

Biogen's Higher SPINRAZA Dose Shows Improved Efficacy in Phase II/III Trial A trial studying a higher dose of Biogen’s spinal muscular atrophy drug SPINRAZA (nusinersen) has met the primary endpoint in a cohort of infants with SMA. The Phase II/III DEVOTE study, which included 145 patients across various ages an...

Aug 23, 2024

Neuromuscular disorders (NMD) encompass a broad spectrum of conditions impacting the peripheral nervous system, which comprises all motor and sensory nerves linking the brain and spinal cord to the body. These disorders comprise various classes of diseases, such as muscular dystrophies, myopathies, motor neuron dis...

Aug 01, 2023

FDA Expands GSK’s Jemperli Approval for Endometrial Cancer GSK plc announced that the US Food and Drug Administration (FDA) has approved Jemperli (dostarlimab) in combination with carboplatin and paclitaxel, followed by Jemperli as a single agent for the treatment of adult patients with mismatch repair deficient...

May 12, 2023

By bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...

Feb 28, 2023

Sage Therapeutics Receives EU Orphan Drug Designation for Huntington's Disease Treatment Candidate Sage Therapeutics declared the European Medicines Agency granted Orphan Drug Designation to SAGE-718 for the treatment of Huntington's disease. SAGE-718 is developing as a potential oral therapy for cognitive disor...

Aug 16, 2022

Novartis’ Canakinumab Fails in Phase III Trials for NSCLC Canakinumab's prospects as an anticancer therapy were already dwindling when a third phase III trial in non-small cell lung cancer (NSCLC) failed to reach its objectives. The most recent setback came from the CANOPY-A study, which included patients with N...

Apr 20, 2022

Drug pricing is one of the hottest topics in the healthcare segment. Several arguments have been put forward by people, governments, healthcare companies, and other organizations in favor of and against expensive medicines. Some of the drugs are so expensive that they are nearly out of reach of the common people. H...

Aug 11, 2020

Roche Set to Compete With Biogen and Novartis in the SMA Market With its First Oral Therapy  The US FDA has recommended the use of Evrysdi (risdiplam), a candidate of Genentech, a Roche company, for spinal muscular atrophy (SMA), an inherited neuromuscular disease resulting in muscle atrophy, in adults and...

Aug 30, 2019

Spinal Muscular Atrophy is a rare and the second most fatal autosomal recessive genetic disorder with an incidence of 1 in 6000 to 1 in 10,000 births. Globally talking, SMA prevalence was reported in 19,343 cases in the year 2017 in the 7MM, and the US accounted for the highest number of cases approximately around ...