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MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Repligen Corporation today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association ("MDA") to support the ongoing development of RG3039 for Spinal Muscular Atrophy ("SMA"). RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.

15 Dec 2010

CSHL scientists reverse symptoms of Type III SMA using ASOs

The devastating, currently incurable motor-neuron disease spinal muscular atrophy might soon be treated with tiny, chemically modified pieces of RNA called antisense oligonucleotides.

13 Jul 2010

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

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

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

Discovery of potential treatment for spinal muscular atrophy

Spinal Muscular Atrophy is the second-leading cause of infant mortality in the world. Ravindra Singh, associate professor in biomedical sciences at Iowa State University's College of Veterinary Medicine, would like to see Spinal Muscular Atrophy lose its high ranking and even slide off the list altogether.

28 Jul 2009

Carrier screening for spinal muscular atrophy should be offered to all couples

Carrier screening for spinal muscular atrophy (SMA) - a serious genetic disease affecting approximately 1 in 10,000 infants that causes progressive muscle weakness and death - should be made available to all families, according to a new practice guideline issued by the American College of Medical Genetics (ACMG).

10 Dec 2008

Potential method for repairing misspelling in DNA code that causes spinal muscular atrophy

Researchers at the University of Delaware have discovered a novel technique - that acts like a "spell-checker" for correcting a misspelling in the DNA code - to repair the defective gene that causes spinal muscular atrophy (SMA).

12 Feb 2008

Indoprofen may lead to new treatments for spinal muscular atrophy

A drug withdrawn from pharmacy shelves over 20 years ago may point the way to a new treatment for spinal muscular atrophy, or SMA, a muscle-wasting and often life-threatening childhood disease.

29 Nov 2004