Experts Agree That a Disease-Modifying Therapy is the Greatest Unmet Need in the Treatment of Duchenne Muscular …

Posted: Published on June 7th, 2012

This post was added by Dr Simmons

BURLINGTON, Mass.--(BUSINESS WIRE)--

Decision Resources, one of the worlds leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that interviewed experts unanimously identify a disease-modifying therapy as the highest unmet need in the treatment of Duchenne muscular dystrophy (DMD). Duchenne is the most common form of muscular dystrophy in children.

Glucocorticoid treatment is the only available therapy for the treatment of DMD, said Decision Resources Analyst Richa Mukherjee, M.Sc., M.Phil., Ph.D. Its a symptomatic therapy that does not target the cause of the disease and its use is often accompanied by concerning side effects. The lack of a disease-modifying therapy to treat DMD presents a high unmet need for drug developers.

According to Niche Markets and Rare Diseases: Muscular Dystrophy, Decision Resources forecasts the launch of four new agents to treat DMD by 2021. These therapies include GlaxoSmithKline/Prosensas GSK-2402968/PRO-051/drisapersen, Santhera/Takeda's Catena/Sovirmas idebenone, AVI BioPharmas eteplirsen/AVI-4658 and Prosensas PRO-044. Of these therapies, GSK-2402968, eteplirsen and PRO-044 are disease-modifying therapies that will heavily saturate their target patient segments however, given the small size of these segments, the market will still be very receptive to novel disease-modifying agents.

The DMD market will grow as a result of polytherapy as patients will be treated with mutation-specific disease-modifying drugs in addition to symptomatic treatments like idebenone, said Dr. Mukherjee. Symptomatic therapies will continue to play a major role for a majority of DMD patients because the new disease-modifying agents target only a subset of the DMD patient population.

About Muscular Dystrophy

Muscular Dystrophy is offered as part of Decision Resources Niche Markets and Rare Diseases service. Each report assesses opportunity in a select drug market across the United States, France, Germany, Italy, Spain and the United Kingdom. The report provides detailed coverage of patient populations, current therapies, unmet needs and emerging therapies, and includes primary research with country-specific thought leaders.

About Decision Resources

Decision Resources (www.decisionresources.com) is a world leader in market research publications, advisory services and consulting designed to help clients shape strategy, allocate resources and master their chosen markets. Decision Resources is a Decision Resources Group company.

About Decision Resources Group

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Experts Agree That a Disease-Modifying Therapy is the Greatest Unmet Need in the Treatment of Duchenne Muscular ...

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