Research News
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January 18, 2012
The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a study designed to identify and validate biomarkers in amyotrophic lateral sclerosis (ALS).
A
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January 15, 2012
Editor's note 2/2/12:This story was updated to reflect the award of a new MDA grant to Eric Sjoberg at Amicus Therapeutics.
Drugs that suppress the immune system can successfully prevent or reverse
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January 13, 2012
Editor's note 2/15/12: On Feb. 15, 2015, Justin Fallon and colleagues announced in the Journal of Neuroscience that they have identified an additional role for biglycan; namely, that it stabilizes
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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 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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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
Update (Feb. 11, 2013): The phase 2 trial is no longer recruiting participants.
Update (March 15, 2012): This phase 1 trial is no longer recruiting participants. A phase 2 trial of sialic acid
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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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November 10, 2011
Editor's note 3/19/12: This story has been updated to reflect that Neuralstem has begun testing its stem cells in the cervical (neck) region of the spinal cord.
Two human clinical trials to test
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November 09, 2011
A large-scale study, supported in part by MDA, seeks to determine the natural history (general disease course) of four subtypes of Charcot-Marie-Tooth disease (CMT), with particular emphasis on
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November 09, 2011
Researchers have found that the drug albuterol appears to be beneficial in two forms of congenital myasthenic syndrome (CMS)— CMS related to mutations in the collagen Q (colQ) gene and CMS related to
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November 08, 2011
Update (March 27, 2013): BioMarin announced March 19, 2013, that it plans to move to a phase 2/3 trial of this drug for late-onset Pompe diseae, pending a review by regulatory authorities. See Pompe
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