Spinal Muscular Atrophy News and Research

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Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of death among infants and toddlers. Characterized by selective loss of nerve cells in the spinal cord, the disease leads to increasing muscular weakness and atrophy. Over time, patients afflicted by SMA continue to lose muscle control and strength, leading to progressive inability to walk, stand, sit up and breathe. It is estimated that approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide with SMA.

Einstein, Montefiore receive NINDS grant to establish clinical site for NeuroNEXT

In a development that could pave the way for treatment for rare neurological diseases and clues to more common ones, physician-scientists at Albert Einstein College of Medicine of Yeshiva University and Montefiore Medical Center, the University Hospital for Einstein, have secured a grant to establish a clinical site for the Network for Excellence in Neuroscience Clinical Trials.

2 Dec 2011

PTC, Roche announce licensing agreement for SMA programme

Roche, PTC Therapeutics, Inc. and the SMA Foundation, announced today a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme.

30 Nov 2011

Targeting synthetic RNA to specific gene could help reduce severity of SMA

A team of University of Missouri researchers have found that targeting a synthetic molecule to a specific gene could help the severity of the disease Spinal Muscular Atrophy (SMA) - the leading genetic cause of infantile death in the world.

22 Nov 2011

SNI receives NINDS grant to conduct clinical research studies on brain disorders

Swedish Neuroscience Institute (SNI) has been selected by the National Institutes of Health as the Pacific Northwest member of a national consortium of 25 neuroscience centers that will conduct clinical research studies on a variety of brain-related diseases.

10 Nov 2011

Cancer cells maintain rapid proliferation due to PK-M2 enzyme

Cancer cells maintain their life-style of extremely rapid growth and proliferation thanks to an enzyme called PK-M2 (pyruvate kinase M2) that alters the cells' ability to metabolize glucose - a phenomenon known as the Warburg effect.

3 Nov 2011

Communication breakdown between nerves and muscles in SMA mouse model

Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy.

27 Sep 2011

Cedars-Sinai inaugurates new facility to generate induced pluripotent stem cells

The Cedars-Sinai Regenerative Medicine Institute has opened a new Induced Pluripotent Stem Cell (iPSC) Core Facility to produce powerful cells capable of making all tissues of the body from adult human skin cells.

23 Sep 2011

New cell type could play an important role in spinal cord injury and disease

The Allen Institute for Brain Science announced today the discovery of a new class of cells in the spinal cord that act like neural stem cells, offering a fresh avenue in the search for therapies to treat spinal cord injury and disease.

16 Sep 2011

Walgreens, MDA team up to offer free seasonal flu shots

Nobody likes to get stuck with a needle. But influenza - the flu - is particularly hazardous for those with neuromuscular disease, which is why the Muscular Dystrophy Association (MDA) and Walgreens have teamed up once again to offer free seasonal flu shots now underway for people who have muscular dystrophy and related diseases.

14 Sep 2011

WSU researcher receives $418,000 NINDS grant to study spinal muscular atrophy

Graham Parker, Ph.D., assistant professor of research in the Department of Pediatrics at Wayne State University's School of Medicine, was awarded $418,000 from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health to study how a particular gene might be involved in the progression of spinal muscular atrophy (SMA) - the number one genetic killer of children younger than 2 years old.

12 Sep 2011

Two ALS-associated genes work in tandem to support long-term survival of motor neurons

Although several genes have been linked to amyotrophic lateral sclerosis (ALS), it is still unknown how they cause this progressive neurodegenerative disease. In a new study, Columbia University Medical Center (CUMC) researchers have demonstrated that two ALS-associated genes work in tandem to support the long-term survival of motor neurons.

2 Sep 2011

MDA announces $13.7M in grants to 40 new muscular dystrophy research initiatives

The Muscular Dystrophy Association today announced funding, totaling $13.7 million, for 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases.

24 Aug 2011

NIH/NINDS awards AMRI to develop pre-clinical drug candidate for spinal muscular atrophy

AMRI today announced it received a federal contract award from the National Institutes of Health (NIH)/National Institute of Neurological Disorders and Stroke (NINDS) to provide chemistry and other drug discovery technologies in support of NINDS' Medicinal Chemistry for Neurotherapeutics Program (MCNP), part of the NIH Blueprint Neurotherapeutics Network.

19 Aug 2011

Repligen first quarter total revenue increases 9% to $7,654,000

Repligen Corporation today reported results for the first quarter of fiscal year 2012, ended June 30, 2011.

27 Jul 2011

New hope for infants born with Spinal Muscular Atrophy

Researchers in Ottawa report new hope for the treatment of infants born with serious genetic disorder.

26 Jul 2011

FDA grants Fast Track designation for Repligen's RG3039 to treat Spinal Muscular Atrophy

Repligen Corporation announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RG3039, a potential treatment for Spinal Muscular Atrophy.

23 Jun 2011

Repligen total revenue increases 30% to $27,291,000 for fiscal year 2011

Repligen Corporation today reported results for the fourth quarter and fiscal year 2011, ended March 31, 2011.

9 Jun 2011

New research on spinal muscular atrophy

Spinal muscular atrophy (SMA) is the leading genetic cause of death in children under 2, with no treatment other than supportive care. In the Proceedings of the National Academy of Sciences, researchers at Children's Hospital Boston show how loss or mutation of the SMA gene causes progressive muscle degeneration and weakness, and suggest a promising approach to treating the condition, sometimes referred to as a "Lou Gehrig's disease of babies."

8 Jun 2011

Evotec, PsychoGenics partner to offer integrated CNS drug discovery solutions

PsychoGenics Inc. today announced that it has entered into a strategic alliance with Evotec AG to provide integrated CNS drug discovery solutions to pharmaceutical and biotech companies.

25 May 2011

Repligen receives approval from FDA to commence RG3039 Phase 1 trial in Spinal Muscular Atrophy

Repligen Corporation announced today that it has received approval from the Food and Drug Administration to initiate a Phase 1 clinical trial of RG3039, a potential treatment for Spinal Muscular Atrophy.

19 May 2011