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SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing
Date:4/24/2012

of dystrophin, the protein missing in the muscle fibers of patients with DMD.

In May 2011, an MDA-supported study showed that daily treatment with SMT C1100 dramatically reduced symptoms in dystrophin-deficient mice with a DMD-like disease. The mice showed reduced muscle abnormalities, increased overall strength and improved ability to resist exercise-related fatigue.

An earlier version of the drug, tested by California-based BioMarin Pharmaceutical, was found safe and well-tolerated in healthy human volunteers, but it failed to reach blood levels considered adequate for therapy.

The reformulated Summit PLC version of the drug is designed to reach adequate blood levels in humans following oral administration.

To learn more, read the Quest News Online article DMD Drug SMT C1100 Moves to Human Testing.

About DMD

DMD is a degenerative muscle disease — affecting boys almost exclusively — that involves progressive degeneration of voluntary and cardiac muscles, with resulting weakness and heart abnormalities. The disease manifests in early childhood, causing delayed motor milestones and, in some cases, cognitive, behavioral or language deficits. Loss of the ability to walk occurs in most children with DMD between the ages of 10 and 12 years; weakened cardiac and respiratory muscles severely limit life span.

The disease is caused by any number of mutations in the X-chromosome gene that carries instructions for the muscle protein dystrophin. Without dystrophin, muscle fibers are abnormally fragile and break down under the stress of contractions.

Corticosteroid medications, such as prednisone and its chemical cousin prednisolone, slow disease progressi
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SOURCE Muscular Dystrophy Association
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Related medicine technology :

1. Halo Therapeutics HT-100 Receives FDAs Orphan Designation for Duchenne Muscular Dystrophy
2. MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy
3. MDA Collaborates with AVI BioPharma on First Phase 2 Placebo-Controlled Clinical Trial of Exon-51 Skipping Drug as Potential Therapy for Duchenne Muscular Dystrophy
4. PTC Therapeutics and Parent Project Muscular Dystrophy Advance Drug Discovery Program to Improve Heart Function in Duchenne/Becker Muscular Dystrophy Patients
5. PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
6. Data Published in Muscle and Nerve Validates the Six-Minute Walk Test as an Outcome Measure in Duchenne Muscular Dystrophy
7. PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy
8. PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy
9. PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
10. PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
11. MDA Awards $1 Million to Tivorsan Pharmaceuticals for Accelerating Pivotal Pre-Clinical Work on TVN-102 as a Potential Muscular Dystrophy Treatment
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