A crucial two-hour meeting between AveXis executives and officials of the U.S. Food and Drug Administration (FDA) will determine the next step for AVXS-101, a spinal muscular atrophy (SMA) treatment that has shown remarkable promise in a Phase 1 trial of babies with SMA type 1 — the most severe form of this muscle-damaging disease.
A new test that can detect the risk of having children with spinal muscular atrophy (SMA) will be commercialized in a joint effort by the two companies, SYNLAB and Counsyl. SYNLAB will offer Counsyl’s proprietary Expanded Carrier Screening (ECS) test under the tradename preconGEN. The test will be offered…
The patient advocacy group Cure SMA will host a webinar May 4 on access to Spinraza (nusinursen), the first drug ever to be approved to treat spinal muscular atrophy (SMA). The webinar will cover several topics, including the current status of Spinraza dosing at U.S. administration sites, commercial and government insurance policies, trial…
A Phase 3 clinical trial evaluating Spinraza (nusinersen) in children with type 2 spinal muscular atrophy (SMA) may play a decisive role in whether private insurers cover the treatment in later-onset SMA patients — those with type 2 and type 3. The study was the focus of an Emerging…
Results from the first clinical trial of AVXS-101, a gene therapy for infants with spinal muscular atrophy (SMA) type 1, reveal dramatically improved survival as well as motor skills in most of the babies involved in the drug’s first clinical trial. Data presented by AveXis at the six-day AAN 2017 Annual Meeting — which…
Spinraza (nusinersen), a treatment for most forms of spinal muscular atrophy (SMA), took a major step forward to becoming available across the European Union, with the Committee for Medicinal Products for Human Use (CHMP) recommending its approval. CHMP is a branch of the European Medicines Agency (EMA), the equivalent of…
Motor milestones — a mark of life quality and potential longevity — are evident in a majority of infants with type 1 spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) in a Phase 3 clinical trial, compared to those who received placebo. New interim trial data, in fact, found that 51 percent of Spinraza-treated infants continue…
Biogen announced the presentation of new data from its current treatments and investigational therapies for neurological and neurodegenerative diseases at the 69th annual meeting of the American Academy of Neurology (AAN) April 22-28 in Boston. The presentations will include new data on Spinraza. Biogen will present data…
Researchers at the Harvard Stem Cell Institute have identified a compound that can restore the SMN motor-neuron protein whose shortage is associated with spinal muscular atrophy (SMA). “This discovery opens up new lines of drug interrogation,” Lee Rubin, the senior author of the study, said in a news story written by Harvard’s Hannah…
Genentech is working on the design of a Phase 3 clinical trial of olesoxime to address U.S. and European regulators’ concerns about the benefits versus the risks of the spinal muscular atrophy (SMA) therapy. Meanwhile, two Phase 2 trials of another Genentech SMA treatment, RG7916, are under way. The U.S.
