“Mo” the Monkey Conducting clinical trials with pediatric patients is always a challenge, as the age of participants adds additional barriers to the successful completion of a scientific study, as well as adds immense stress on both the child and parents. This is particularly true with Type 1 Spinal Muscular…
Pfizer and Repligen have ended their partnership focused on developing therapeutic options for the treatment of spinal muscular atrophy (SMA), according to a news report from GEN and other news outlets. The collaboration was worth up to $70 million, but was ended by Pfizer…
Albany Molecular Research (AMRI), a contract research and manufacturing organization that provides drug discovery, development, cGMP manufacturing and aseptic fill and finish to the pharmaceutical and biotechnology industries, recently announced it has been granted a 10-year federal contract award care of the National Institutes of Health (NIH) to secure their drug development…
A team of researchers from Denmark found that a cycle training program was able to modestly improve oxidative capacity in patients with Spinal Muscular Atrophy type III. Spinal muscular atrophy (SMA) is a motor neuron disease characterized by an autosomal recessive disorder that is caused by inherited mutations in the survival motor neuron…
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…
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…
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