Muscular Atrophy News and Research RSS Feed - Muscular Atrophy News and Research

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin. [More]
Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received new awards from the California Institute of Regenerative Medicine (CIRM), the state stem cell research agency, that will forward revolutionary stem cell science in medicine. [More]
Joint research program in SMA enters initial stage of clinical development

Joint research program in SMA enters initial stage of clinical development

PTC Therapeutics, Inc., the SMA Foundation, and Roche, announced today that their joint research program in Spinal Muscular Atrophy has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. [More]
Combination of NGF microspheres and chitosan conduits exhibit better effects in repairing facial nerve injury

Combination of NGF microspheres and chitosan conduits exhibit better effects in repairing facial nerve injury

The chitosan molecule can promote nerve cell adherence and growth along the surface of the material. It can enhance the adherence and influx of Schwann cells, thus encouraging the growth of axons. [More]
NUS scientists open possible new route for treatment of spinal muscular atrophy

NUS scientists open possible new route for treatment of spinal muscular atrophy

A recent study led by scientists from the National University of Singapore (NUS) opens a possible new route for treatment of Spinal Muscular Atrophy (SMA), a devastating disease that is the most common genetic cause of infant death and also affects young adults. [More]
Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

The exact definition of orphan diseases varies depending on where you are in the world. In the EU it's defined as a prevalence of less than 1 in 2,000, which equates to about 250,000 patients across the EU. [More]
Principal investigators at Nationwide Children's Hospital elected as AAAS Fellows

Principal investigators at Nationwide Children's Hospital elected as AAAS Fellows

Brian K. Kaspar, PhD, and Veronica J. Vieland, PhD, principal investigators in The Research Institute at Nationwide Children's Hospital, have been named Fellows of the American Association for the Advancement of Science (AAAS). [More]
Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy. [More]
Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children's Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA). [More]
Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and the Muscular Dystrophy Association (MDA) today announced a new partnership to develop and implement the U.S. Neuromuscular Disease Registry, a patient registry that will play an important role in determining effective treatments for people with muscular dystrophy and related muscle diseases. [More]
ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). [More]
Final patient completion of phase II study of TR040303 in patients treated for acute myocardial infarction announced by Trophos

Final patient completion of phase II study of TR040303 in patients treated for acute myocardial infarction announced by Trophos

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today the final patient completion of a phase II study of TR040303 in patients treated for acute myocardial infarction (MI). Outcome data of this study is expected to be available towards the end of 2013. [More]
Stem cell lines are ideal research tools for designing models to understand disease progression

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA. [More]
Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug. [More]
Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

Researchers develop TSUNAMI method to create animal model of adult-onset version of SMA

A research team at Cold Spring Harbor Laboratory has used a recently developed technology they call TSUNAMI to create the first animal model of the adult-onset version of spinal muscular atrophy, a devastating motor-neuron illness. [More]
One-time treatment slows onset and progression of ALS, study finds

One-time treatment slows onset and progression of ALS, study finds

Studies of a therapy designed to treat amyotrophic lateral sclerosis suggest that the treatment dramatically slows onset and progression of the deadly disease, one of the most common neuromuscular disorders in the world. [More]
Biogen Idec And Isis Ink Fourth Deal In Two Years

Biogen Idec And Isis Ink Fourth Deal In Two Years

... [More]
Neurodyn acquires all assets related to new drug candidate Memogain from Galantos Pharma

Neurodyn acquires all assets related to new drug candidate Memogain from Galantos Pharma

Neurodyn Inc. announced today that it has acquired from Galantos Pharma GmbH, Mainz, Germany all assets related to their Alzheimer's prescription drug candidate - Memogain. Memogain has completed an extensive preclinical development program and is ready to proceed into Phase 1 clinical trials in late 2013. The terms of the acquisition were not disclosed. [More]

Repligen Earns $1M Pfizer Milestone Payment

... [More]
Medical investigators discover new aspect of gene-splicing process that produces mRNA

Medical investigators discover new aspect of gene-splicing process that produces mRNA

A rare, small RNA turns a gene-splicing machine into a switch that controls the expression of hundreds of human genes. Howard Hughes Medical Institute Investigator and professor of Biochemistry Gideon Dreyfuss, PhD, and his team from the Perelman School of Medicine at the University of Pennsylvania, discovered an entirely new aspect of the gene-splicing process that produces messenger RNA (mRNA). [More]