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Specials and orphan drugs
Specials and orphan drugs
Positive CHMP opinion for Akcea orphan drug inotersen
Orphan drug designation for first-in-class treatment for C3 glomerulopathy
Letermovir granted marketing authorisation as an orphan medicinal product
Abeona Therapeutics Receives Orphan Drug Designation in EU for ABO-102
Orphan drug designation granted to nintedanib for treatment of systemic sclerosis
Dompé’s rhNGF receives orphan drug designation from the EMA
Adcetris® is the first ultra-orphan medicine recommended by AWMSG via the new assessment process
Albireo granted orphan drug designation for A4250
Issues affecting access to orphan drugs in Europe
Market access of orphan drugs: one size fits all?
COMP adopts applications for four lipodystrophy drugs
Eculizumab recommended for orphan drug status
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