Muscular Atrophy News and Research

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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).

4 Oct 2013

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.

2 Oct 2013

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.

25 Sep 2013

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.

19 Sep 2013

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.

10 Sep 2013

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.

10 Sep 2013

Biogen Idec And Isis Ink Fourth Deal In Two Years

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9 Sep 2013

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.

5 Sep 2013

Repligen Earns $1M Pfizer Milestone Payment

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4 Sep 2013

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).

22 Aug 2013

Research: RG3039 drug can extend survival, improve function in spinal muscular atrophy mouse models

In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and improve function in two spinal muscular atrophy (SMA) mouse models.

5 Jun 2013

CGCF backs Canadian scientist's research on Spinal Muscular Atrophy

The Canadian Gene Cure Foundation, in partnership with the Canadian Institutes of Health Research through its Institute of Genetics, is pleased to announce the awarding of a $90,000 Champions of Genetics: Building the Next Generation Grant to Dr. Faraz Farooq, a Scientist at the Children Hospital of Eastern Ontario Research Institute.

9 May 2013

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.

24 Apr 2013

Clinical trial signals new era in treatment of neurodegererative disorders

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.

4 Apr 2013

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.

21 Mar 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.

18 Mar 2013

Phase 1b study of olesoxime initiated in multiple sclerosis (MS) patients

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14 Mar 2013

Researchers identify new mechanism in onset of motor neuron diseases

The British astrophysicist Stephen Hawking is likely to be the world's most famous person living with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. ALS is a progressive disease affecting motor neurons, nerve cells that control muscle function, and nearly always leads to death.

11 Mar 2013

Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

1 Mar 2013

Quest Diagnostics offers access to a new non-invasive prenatal test developed by Natera

Quest Diagnostics (NYSE:DGX), the world’s leading diagnostic information services company, and Natera, a leading innovator in prenatal genetic testing, today announced that Quest Diagnostics will offer physicians access to Panorama™, a new non-invasive prenatal test developed by Natera.

From Quest Diagnostics 21 Feb 2013