FDA turns down Amryt’s epidermolysis bullosa drug
Amryt Pharma, dermatology, epidermolysis bullosa, inherited disease, rare disease, regulatory
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inherited disease
Applied Therapeutics slumps as FDA seeks more data on lead drug
Applied Therapeutics, galactosaemia, inherited disease, rare disease, regulatory
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Out of the darkness: new gene therapies for eye diseases
eye disease, gene therapy, inherited disease, patient perspective, rare disease, Spark Therapeutics
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US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.