Image pharmaphorum Editor R&D A lifeline for rare disease patients For more than 25 years Alexion has been blazing a trail in rare diseases, bringing a series of life-changing medicines to market. R&D Living with LHON: Adapting to sight loss at 15 years old When faced with a devastating diagnosis, a family has two choices – to give up or make it their mission to adapt and carry on. R&D How pharma can support doctors who treat rare diseases Where do doctors see patients with rare diseases, how do they keep up with treatment advances and what can pharmaceutical companies do to help? R&D The patient perspective on advocacy Growing up with the shadow of Huntington’s disease (HD) means Seth Rotberg understands the importance of peer support and credible information. Load more results
R&D A lifeline for rare disease patients For more than 25 years Alexion has been blazing a trail in rare diseases, bringing a series of life-changing medicines to market.
R&D Living with LHON: Adapting to sight loss at 15 years old When faced with a devastating diagnosis, a family has two choices – to give up or make it their mission to adapt and carry on.
R&D How pharma can support doctors who treat rare diseases Where do doctors see patients with rare diseases, how do they keep up with treatment advances and what can pharmaceutical companies do to help?
R&D The patient perspective on advocacy Growing up with the shadow of Huntington’s disease (HD) means Seth Rotberg understands the importance of peer support and credible information.
News EU clears first drug therapy for rare disease cALD Neuraxpharm and Minoryx' Nezglyal has become the first approved drug therapy in the EU for rare neurodegenerative disease cALD.
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