Spinal muscular atrophy
Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy.
Genentech announced positive topline results from its pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 SMA.
The company has suggested that it plans to undercut both Biogen and Novartis on price in order to make up for being third-to-market.
Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people living with Types 1, 2 or 3 SMA
Genentech announced this morning that its experimental SMA drug risdiplam hit the mark in the second part of the pivotal SUNFISH study of patients with Type 2 and 3 of the disease.
BillionToOne and Eluthia have announced the launch of the first and only non-invasive prenatal test, allowing to screen for the autosomal-recessive conditions cystic fibrosis, spinal muscular atrophy, sickle cell disease and other hemoglobinopathies.
While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy.
AveXis presented new interim data from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1.
Catalyst Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, announced its support of Spinal Muscular Atrophy Awareness Month.
Novartis Chief Executive Officer Vas Narasimhan is excited about the company’s progress through the first half of 2019 and expects that momentum to continue with a “catalyst-rich” second half of the year.
PRESS RELEASES