Muscular Atrophy News and Research

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Spinal muscular atrophy screening, not cost effective

In a study to be presented today at the Society for Maternal-Fetal Medicine's (SMFM) annual meeting, The Pregnancy Meeting -, in Chicago, researchers will unveil findings that show that it is not cost effective to screen for spinal muscular atrophy.

5 Feb 2010

CSC, ALS TDI extend collaboration in advancing potential stem cell therapy for ALS

California Stem Cell, Inc. (CSC) and ALS Therapy Development Institute (ALS TDI) are pleased to announce an extension and expansion of their collaboration aimed at advancing a potential stem cell therapy for ALS (amyotrophic lateral sclerosis). This effort will build on work that has already been completed as part of this on-going partnership to understand how stem cells, and their derivatives, may be applied to treatment of this fatal neurodegenerative disease.

3 Feb 2010

iPierian receives UK patent for induced pluripotent stem cell technology

iPierian, Inc., a biopharmaceutical company focused on changing the paradigm of drug discovery and development through the application of cellular reprogramming, today announced that the Intellectual Property Office in the United Kingdom has issued a Notification of Grant to iPierian for UK patent No. GB2450603, its first patent related to induced pluripotent stem cell (iPSC) technology.

29 Jan 2010

Simple, five minute saliva test to determine baby's risk for more than 100 life-threatening genetic diseases

Genetic diseases like those seen in the new Harrison Ford movie "Extraordinary Measures" can now be prevented with a simple saliva test which is free with insurance for more than 100 million Americans.

22 Jan 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010

A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 Jan 2010

CSC completes a Series B financing round

California Stem Cell, Inc. (CSC), a leading stem cell company, announced that it has completed a Series B financing round with private investors. The financing will enable the company to support the advancement of its therapeutic products to human clinical trials, as well as the generation and distribution of its tools to aid in stem cell research and drug discovery.

21 Dec 2009

CSC's stem cell-derived motor neuron product for SMA receives FDA orphan drug designation

California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that the FDA has granted orphan drug designation to MOTORGRAFT™, a stem cell-derived motor neuron product, for the treatment of Spinal Muscular Atrophy (SMA).

9 Dec 2009

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

Isis Pharmaceuticals, Inc. announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.

4 Dec 2009

DFG to strengthen its support for young researchers, approves establishment of 11 new Research Training Groups

The Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) has intensified its efforts to strengthen its support of young researchers. At its fall session in Bonn, the responsible Grants Committee of Germany's central research funding organisation approved the establishment of eleven new Research Training Groups. In these groups, doctoral researchers can earn their doctorates in a structured research and qualification programme.

13 Nov 2009

Second-quarter fiscal 2010 results announced by Repligen

Repligen Corporation today reported results for the second quarter of fiscal year 2010, ended September 30, 2009. Total revenue for the quarter was $5,421,000 compared to total revenue of $5,090,000 for the second quarter of fiscal year 2009 ended September 30, 2008.

5 Nov 2009

Tetracycline-like compound raises hope for treatment of spinal muscular atrophy

A chemical cousin of the common antibiotic tetracycline might be useful in treating spinal muscular atrophy (SMA), a currently incurable disease that is the leading genetic cause of death in infants. This is the finding of a research collaboration involving Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory (CSHL) and scientists from Paratek Pharmaceuticals and Rosalind Franklin University of Medicine and Science.

5 Nov 2009

Families of Spinal Muscular Atrophy and Repligen partner to develop a treatment for SMA

Families of Spinal Muscular Atrophy (FSMA) announced today that it has entered into a groundbreaking exclusive license agreement with Repligen Corporation for the development of a potential treatment of Spinal Muscular Atrophy (SMA).

23 Oct 2009

Intellectual property covering compounds that have utility in treating SMA licensed

Repligen Corporation announced today that it has entered into an exclusive license agreement with Families of Spinal Muscular Atrophy (FSMA) for intellectual property covering compounds which may have utility in treating Spinal Muscular Atrophy (SMA).

23 Oct 2009

iPierian to participate in $3.7 million NIH grant awarded to The Johns Hopkins University

iPierian, Inc., a biopharmaceutical company focused on changing the paradigm of drug discovery and development through the application of cellular reprogramming, today announced that it has been selected to participate in a $3.7 million National Institutes of Health (NIH) Grand Opportunities (GO) grant awarded to The Johns Hopkins University.

22 Oct 2009

Multi-million dollar cooperative agreement for SMA drug development program

Paratek Pharmaceuticals (Boston, MA) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announced today that a jointly funded drug development program for Spinal Muscular Atrophy (SMA) has been awarded a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS).

19 Oct 2009

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.

9 Sep 2009

Psychogenics and Astrazeneca sign agreement to identify new compounds for treating Central Nervous System disorders

PsychoGenics Inc. and AstraZeneca have entered into a drug discovery and development agreement to identify compounds that are likely to be useful for the treatment of certain Central Nervous System (CNS) disorders.

31 Aug 2009

Office of Orphan Products Development of the FDA grants Orphan Drug Designation to Quinazoline495

Families of Spinal Muscular Atrophy (Libertyville, IL) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted Orphan Drug Designation to Quinazoline495 for the treatment of Spinal Muscular Atrophy.

29 Aug 2009

Promising new treatment target for Kennedy's disease

New research with transgenic mice reveals that a therapy directed at the muscle significantly improves disease symptoms of a genetic disorder characterized by destruction of the neurons that control movement. The study, published by Cell Press in the August 13th issue of the journal Neuron, highlights a promising new treatment for this currently incurable and nontreatable neurodegenerative disorder.

12 Aug 2009