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10 companies driving forward the rare disease market

Authored by Liftstream

In recognition of Rare Disease Day 2013 Liftstream has chosen to profile 10 highly innovative biotech companies which are highly active in rare disease research and development. The companies analysed range from those with marketed products to companies just starting on the clinical development programme.  To coincide with the launch of Liftstream’s case study on Duchenne Muscular Dystrophy (DMD) which will be released in the coming days, we have chosen 4 DMD specialist companies as part of our analysis: Sarepta Therapeutics, Prosensa, PTC Therapeutics and Summit plc.

The rare disease market has gained significant attention in recent times and now attracts interest from many large pharmaceutical companies and investors who identify the importance of these therapies and the commercial benefits from investing in rare diseases. There are currently an estimated 7000 rare diseases and only 75 approved therapies approved in Europe, showing there is considerable work still to be done in getting new drug to market. Therapies aside, there is still very much to do to improve the standard of care for patients affected by rare diseases.

The companies Liftstream profiled are:

Sangart awaits key clinical data in 2013

Amicus Therapeutics looking to bounce back in 2013

Aegerion brings new hope to HoFH patients

Oxyrane seeking to advance Pompe Disease Programme in 2013

Ultragenyx focused on ultra-rare disease future 

Funding success allow Promethera Biosciences to look to clinical data in 2013

Synageva offering LSD patients renewed hope

Sarepta hopes high for first Duchenne (DMD) therapy 

Prosensa & GSK pool resources to drive Drisapersen in Duchenne

PTC Therapeutics opts for conditional approach in race to market DMD drug

If you wish to discuss any of the profiles further please contact Liftstream at or tweet @liftstream

Liftstream is supporting Rare Disease Day 2013. In profiling these companies we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter

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