Category Archives: News from Other Sources

News gathered from external sources by our RSS aggregator.

Companies Expand Development of Exon Skipping for DMD

AVI BioPharma is developing two new exon-skipping drugs; GlaxoSmithKline will conduct more trials of GSK2402968, for which participants are being recruited.

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BioMarin Seeking US Approval of LEMS Drug

A drug approved in Europe to treat Lambert-Eaton myasthenic syndrome is now being tested in the United States; clinical trial participants sought.

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ALS TDI Summit: ‘An Incredibly Hopeful Time’

ALS TDI’s 2011 Leadership Summit included the latest on ALS research and a discussion of speeding the pace of drug development.

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CoQ10, Lisinopril Studied in DMD, BMD, LGMD2C-2F, LGMD2I

Researchers at six centers are studying possible cardiac benefits of dietary supplement coenzyme Q10 and cardiovascular drug lisinopril in three MD forms.

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ALS Clinical Trials Briefs: Cytokinetics, Neuralstem Advance

CK-2017357 is being tested in combination with riluzole, and Neuralstem will test delivery of its stem cells into the cervical region of the spinal cord.

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Researchers studying CMT1B, CMT2A, CMT4A, CMT4C, Others

A multinational study supported by MDA and NINDS will study correlations between mutations and symptoms in four subtypes of Charcot-Marie-Tooth disease.

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Albuterol Improved Quality of Life in Two Forms of CMS

An MDA-supported open-label trial found the asthma drug benefited 17 of 18 people with either of two forms of congenital myasthenic syndrome.

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BioMarin Testing Drug in Late-Onset Pompe Disease

Clinical trial testing experimental enzyme replacement drug in people with Pompe disease who are at least 13 years old.

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Experimental Drug Seems to Slow Respiratory Decline in DMD

New data show that Catena (idebenone) has a modest effect on respiratory function, bolstering results from an earlier trial.

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Myasthenia Gravis Trials Now Open

Two separate trials will test the experimental drug CK-2017357 and a surgical procedure called a thymectomy in people with MG.

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