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Moving Toward 'Next-Generation' Gene Therapy

MDA partnered with a French muscle disease association to host a symposium on advancing gene therapy for neuromuscular diseases

posted on May 23, 2013 - 5:00am
Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).
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FA: RG2833 is Well-Tolerated and Increases Frataxin Gene Activity

Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia

posted on May 17, 2013 - 4:39pm
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.
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Newborn Screening Recommended for Pompe Disease

Federal committee votes to add the enzyme deficiency disorder to the list of recommended conditions for which states screen newborns

posted on May 17, 2013 - 2:45pm
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a list of diseases that it recommends states screen for in newborns.
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DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug

PTC Therapeutics' large-scale multinational trial of ataluren for nonsense-mutation Duchenne or Becker MD has opened its first site in Cincinnati, Ohio

posted on May 8, 2013 - 10:31am
A large-scale, multinational, phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.
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SMA: Trial Tests Antisense Therapy in Infants

Isis Pharmaceuticals has launched a phase 2 trial to test its experimental antisense drug ISIS-SMNRx in infants with spinal muscular atrophy

posted on May 7, 2013 - 12:15pm
A phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) has opened at four trial sites in the United States and Canada. Enrollment is expected to begin soon.
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MTM Phone-Based 'Event' Study Now Open

Researchers conducting a study of medical changes and complications in myotubular myopathy (MTM) are seeking individuals with MTM or their parents for 12 monthly phone interviews

posted on May 3, 2013 - 11:05am
The Myotubular Myopathy Event Study, a telephone-based survey, will gather information about MTM-associated events, such as emergency room visits, hospitalizations, medication reactions, and complications from medical procedures, as well as improved or declined motor and respiratory function.
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UPDATE: ACE-031 Clinical Trials in Duchenne MD

In early 2011, clinical trials of ACE-031, a myostatin inhibitor, were halted pending resolution of safety issues; in May 2013, the developers discontinued work on the drug

posted on May 2, 2013 - 12:00pm
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