alexa Advanced techniques for Muscular Dystrophy

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Advanced techniques for Muscular Dystrophy

Therapy development processes is among the various types of muscular dystrophy, basic scientists have had variable success in the identification of disease mechanisms that can serve as novel targets for drug development. Duchenne muscular dystrophy (DMD) is target-rich (such as fibrosis, membrane repair, and dystrophin replacement)-it is unclear which targets represent the best opportunities and how the drugs and biologics that emerge will be best combined to effectively manage patients. In this target-rich environment, with limitations on time, effort and funding, participants discussed the critical question as to when enough was known about a particular target and candidate therapeutic to enter into a formal therapy development program. By contrast, the pathogenic mechanisms responsible for myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are just starting to emerge and current research efforts are either on basic science focused upon understanding disease mechanisms or on drug discovery focused on general interventions that may be common to several of the muscular dystrophies (such as enhancing strength of remaining muscle fibers). There were clear reasons for optimism-as recent progress in mechanistic understanding of FSHD and DM offer hope for the initiation of targeted therapeutic development efforts for these diseases. The Challenges of Target Identification and Development Decisions Working Group reviewed the known mechanistic targets in muscular dystrophy, potential strategies to attack these targets and examined the question of when in the course of disease knowledge acquisition to launch a therapy development program.

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