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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

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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

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

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. [More]

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. [More]

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. [More]
Clinical trial signals new era in treatment of neurodegererative disorders

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. [More]

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. [More]
Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

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. [More]
Phase 1b study of olesoxime initiated in multiple sclerosis (MS) patients

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

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Researchers identify new mechanism in onset of motor neuron diseases

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. [More]

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). [More]
Quest Diagnostics offers access to a new non-invasive prenatal test developed by Natera

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. [More]

Counsyl, ARUP partner to expand pregnancy genetic test offering for prospective parents

Counsyl has partnered with ARUP Laboratories, a nonprofit enterprise of the University of Utah, to expand its pre-pregnancy and early pregnancy genetic test offering for prospective parents. ARUP's molecular genetic testing laboratory already provides a comprehensive set of testing options for a broad range of inherited disorders. [More]
Researchers to develop new diagnostic tools and treatments for people with rare diseases

Researchers to develop new diagnostic tools and treatments for people with rare diseases

A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in this area on a global scale. [More]

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA). [More]

Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Biogen Idec and Isis Pharmaceuticals, Inc. today announced that they have entered into a global collaboration agreement under which the companies will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders. [More]