The U.S. Food and Drug Administration (FDA) has granted Cytokinetics’ investigational spinal muscular atrophy (SMA) treatment CK-2127107 Orphan Drug Designation. That gives it seven years of U.S. marketing exclusivity, tax credits for clinical research and a waiver from FDA user fees. CK-2127107, developed in partnership with Astellas Pharma, is…
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…
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 Science…
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…
Nearly half of young people with spinal muscular atrophy (SMA) — and especially those who are underweight — have abnormally high levels of leptin, a metabolic hormone linked to both disease severity and limited mobility, says a new study. The findings show that SMA is associated with abnormal metabolic processes, and researchers suggests…
Children with spinal muscular atrophy (SMA) have poor bone health that leads to common thigh-bone fractures, regardless of the state of their disease, according to a study. Although few of the children could be classified as having osteoporosis, researchers noted that osteoporosis criteria were developed for healthy children. That means the…
Fetuses with the most severe form of spinal muscular atrophy (SMA) usually die in the womb, but a newborn with the condition survived 12 days, according to a case study. Infants born with SMA type 0 have mutations of the SMN gene. Since the newborn had fewer copies of the SMN2…
An analysis of U.S. orphan drug designations and approvals suggests that current incentives, detailed in the Orphan Drug Act (ODA) of 1983, have failed to stimulate the development of orphan drugs targeting spinal muscular atrophy (SMA) and other rare diseases. Rather, researchers at the University of Massachusetts-Amherst suggest that the…
Researchers may finally have figured out why a loss in the SMN protein that is crucial for all cells so quickly affects motor neurons, leading infants to develop spinal muscular atrophy (SMA). SMN aids in the transport of certain molecules in all cells, processing RNA, and the mutation-caused drop in its levels within cells…
Lowering levels of a newly identified protein, in combination with a splice-modifying therapy, improved symptoms and survival in animal models of spinal muscular atrophy (SMA), according to a recent study. The finding suggests that targeting the factor — called neuronal calcium sensor neurocalcin delta (NCALD) — could be beneficial for SMA patients,…
