Shown here are spinal sections from three different mice with spinal muscular atrophy. Systemic drug treatment (middle panel) increases the presence of motor neurons (red spots) over the untreated mice (left panel). Surprisingly, the results are very similar when treatment is excluded from the central nervous system…
News
A recent review explored the current evidence regarding the genetic mechanisms implicated in Spinal Muscular Atrophy and its potential applicability for the development of treatments. The review entitled “The Genetics of Spinal Muscular Atrophy: Progress and Challenges” recently published in the journal Neurotherapeutics, was conducted…
Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA) — a severe and devastating neuromuscular disease that is one of the most common fatal genetic disorders, occurring in 1 in every 6,000 to 10,000 live births and the number-one genetic cause of infant death…
A study published by a team of researchers at the Cold Spring Harbor Laboratory (CSHL) suggests that a drug to treat spinal muscular atrophy (SMA) in a mouse model is effective when administered subcutaneously in peripheral tissue and not just in the central nervous system (CNS). This important…
Swiss global health-care company Roche announced just last week that it has purchased Trophos, a privately-owned Marseille, France-based clinical stage pharmaceutical company that specializes in mitochondrial targeted compounds that work to improve the function and survival of stressed cells by preventing mitochondrial permeability transition, hence helping prevent cell death. Roche has taken an interest in…
Researchers from the University of Pennsylvania and Columbia University Medical Center in New York have studied the characteristics of spinal muscular atrophy (SMA) type I in five infants with the disorder in a study titled Spectrum of neuropathophysiology in spinal muscular atrophy type I published in the Journal of…
Carlsbad, California-based Isis Pharmaceuticals, Inc. has received a $7 million milestone payment from Biogen Idec, to advance an ongoing open-label extension clinical study of the drug ISIS-SMNRx in children with spinal muscular atrophy (SMA). The study of ISIS-SMNRx is open to children with SMA who have completed…
A recent study identified several key health-related factors as the most concerning issues for both patients/families and healthcare professionals that deal with Spinal Muscular Atrophy. The study, entitled “Assessing the Needs of the SMA Population: Survey Results of Health Care Providers and Families,” was recently published in the…
2014 National Goodwill Ambassador announcement at Harley-Davidson headquarters, in Milwaukee Nine-year-old Reagan Imhoff has been chosen as the 2015 National Goodwill Ambassador, after spending 2014 traveling across the country to give a face and a voice to the Muscular Dystrophy Association (MDA). Despite the difficulties of suffering from…
Dr. Barrington G. Burnett was named the recipient of the 2014 Emerging Investigator $62,500 Finalist Award, according to a recent Gwendolyn Strong Foundation and FightSMA announcement. Dr. Barrington G. Burnett’s project was entitled “Targeting the Ubiquitin Proteasome System to Treat Spinal Muscular Atrophy,” with the goal of identifying pathways and genes that…
