Category Archives: News from Other Sources

News gathered from external sources by our RSS aggregator.

Is Prenatal Screening For Rare Diseases Like Spinal Muscular Atrophy Too Costly?

Spinal Muscular Atrophy (SMA) is one of many serious disorders for which prenatal testing is available. SMA affects approximately 1 in 10,000 live births and is the leading genetic cause of infant mortality and the second most common autosomal recessive disorder, after cystic fibrosis…

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Gene Therapy Book by MDA Grantee

‘Muscle Gene Therapy’ brings together the latest research in this promising field.

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ALS TDI: Full Speed Ahead

ALS TDI’s first-quarter 2010 research update can be summed up with one word: momentum.

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ISIS Initiates Phase 1 Clinical Trial Of ISIS-SOD1Rx In Patients With ALS

Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced that it has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig’s disease also known as familial amyotrophic lateral sclerosis (ALS). Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1…

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ALS SOD1 Trial: A ‘Watershed Moment’

Human testing has begun of ISIS-SOD1-Rx, a compound designed to block production of a toxic protein in people with the SOD1-related form of familial (inherited) ALS

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ALS SOD1 Trial: a ‘Watershed Moment’

Human testing has begun of ISIS-SOD1-Rx, a compound designed to block production of a toxic protein in people with the SOD1-related form of familial (inherited) ALS

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DMD/BMD Research: Ataluren Results Disappointing

The experimental drug ataluren, developed to overcome nonsense mutations in Duchenne and Becker MD, did not meet its primary end point in a large-scale human trial.

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Gene Therapy Rescues SMA Mice

A research team reports “unprecedented” improvement in newborn SMA-affected mice that received gene therapy via intravenous injection.

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Gene Therapy Rescues Mice

A research team reports “unprecedented” improvement in newborn SMA-affected mice that received gene therapy via intravenous injection.

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A Quicker Trip

Two federal agencies, the NIH and FDA, are cooperating in an attempt to reduce the time it takes to approve new drugs.

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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...
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