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10 companies driving forward the rare disease market

Authored by Liftstream

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PTC Therapeutics opts for conditional approach in Duchenne

Authored by Paul Foster

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Prosensa & GSK pool resources to drive Drisapersen in Duchenne

Authored by Karl Simpson

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Sarepta’s hopes high for first Duchenne (DMD) therapy

Authored by James Sheppard

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Synageva offering LSD patients renewed hope

Authored by James Sheppard

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Funding successes allow Promethera to look to clinical data in 2013

Authored by James Sheppard

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Ultragenyx focused on ultra-rare disease future

Authored by Paul Foster

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Oxyrane seeking to advance Pompe Disease programme in 2013

Authored by James Sheppard

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Aegerion brings new hope to HoFH patients

Authored by Karl Simpson

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Roche Snags Phase III Agromegaly Drug from Chiasma

Roche have announced they have acquired the rights to a phase III oral Agromegaly drug Octreolin from, Israeli biotech, Chiasma. Agromegaly is a rare progressive disease caused by the over production of growth hormone resulting in severe disfigurement. Agromegaly is estimated to occur in approximately 6 in every 100,000 people.

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