Research News
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January 11, 2012
A January 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the potential of a strategy called exon skipping as a treatment for Duchenne muscular dystrophy (DMD).
The
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January 03, 2012
The U.S. Food and Drug Administration approved the NeuRx Diaphragm Pacing System as a humanitarian use device (HUD), a designation given to medical devices intended for use in rare diseases. For
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January 01, 2012
A 24-person, phase 1 trial to test the safety and tolerability of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at Columbia University Medical Center in
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January 01, 2012
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported,
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January 01, 2012
Stem cells have been much in the news lately, including for neuromuscular diseases.
One extremely promising approach has been to create stem cells from the adult (differentiated) cells of humans or
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January 01, 2012
Dexpramipexole shows efficacy in ALS
Newly published data confirm that in a two-part phase 2 clinical trial, the experimental therapy dexpramipexole showed dose-related slowing of symptom progression
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January 01, 2012
One of the first things that comes with a diagnosis of ALS is the determination of which “form” a person has: familial or sporadic.
Sometimes it appears obvious. If there’s a known history of the
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January 01, 2012
Researchers studying ‘natural history’ of four CMT subtypes
Participants are sought for a large-scale study, supported in part by MDA, to determine the natural history (general disease course) of
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January 01, 2012
Fight smart, not just hard. That’s the motto of Richard Bedlack, a world-renowned ALS research warrior who directs the ALS Clinic at Duke University, Durham, N.C. “Everyone I’ve ever met who is
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December 28, 2011
The Muscular Dystrophy Association has awarded a $278,850 grant to the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., to support testing of a mouse version of a compound called
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December 25, 2011
A research team has demonstrated that a cell-penetrating molecule called TAT transported human frataxin protein to its proper place in cells, where it normalized growth, improved heart structure and
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December 21, 2011
The ALS Therapy Development Institute (ALS TDI), an MDA-supported, nonprofit biotechnology organization dedicated to developing effective treatments for amyotrophic lateral sclerosis (ALS), will
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December 20, 2011
MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment ofDuchenne muscular dystrophy (DMD). Summit is an Oxford,
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December 19, 2011
Update (June 27, 2012): Amicus Therapeutics announced June 26, 2012, that preliminary results are encouraging from this phase 2 trial of AT2220 with enzyme replacement therapy for Pompe disease. For
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December 14, 2011
A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular
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December 13, 2011
In the first stage of an ongoing phase 2 clinical trial to test the experimental therapy CK-2017357 in amyotrophic lateral sclerosis (ALS), the drug was found to be safe and well-tolerated.
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December 08, 2011
The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and
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November 30, 2011
Swiss biotechnology company Roche, and PTC Therapeutics in South Plainfield, N.J., on Nov. 29, 2011, announced a collaboration through which the two companies will work together to advance drug
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November 30, 2011
Editor's note (March 15, 2012): This phase 1 trial is no longer recruiting participants. A phase 2 trial of sialic acid extended release tablets in people with GNE-related IBM is getting under way.
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November 28, 2011
As part of the upcoming International Myotonic Dystrophy Consortium (IDMC-8) in Clearwater, Fla., a session will be held Saturday, Dec. 3, 2011, to provide a research update for individuals and
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November 23, 2011
Newly published data confirm that in a two-part phase 2 clinical trial, the experimental therapy dexpramipexole showed dose-related slowing of symptom progression and increased survival time in
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November 20, 2011
Two pharmaceutical companies recently announced they are expanding their development and testing of exon-skipping drugs for Duchenne muscular dystrophy (DMD).
Exon skipping is an experimental
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November 17, 2011
BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridinephosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic
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November 16, 2011
ALS experts talked about the state of research, and industry leaders discussed ways to improve the pace and efficiency of ALS drug development at the 2011 ALS TDI Leadership Summit, Nov. 4, 2011. The
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November 14, 2011
Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in
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