Rare diseases
Pfizer axes development of Duchenne muscular dystrophy antibody
Piotr Wnuk
DMD, Duchenne, Duchenne muscular dystrophy, Pfizer, rare disease
0 Comment
US regulators reject Akcea and Ionis rare disease drug
Piotr Wnuk
Akcea Therapeutics, familial chylomicronemia syndrome, Ionis Pharmaceuticals, rare disease, Waylivra
0 Comment
AstraZeneca/ Merck Inc/ News/ News/ News/ Oncology/ R&D/ Rare diseases/ Top stories/ UK & Europe
Neurofibromatosis treatment granted orphan status by EMA
Piotr Wnuk
ASCO 2018, AstraZeneca, ema, genetic, Merck & Co, Neurofibromatosis, NF1, Oncology, Selumetinib
0 Comment
Market Access/ News/ News/ News/ News/ Patients/ R&D/ Rare diseases
The first lipodystrophy treatment for European patients
Piotr Wnuk
Lipodystrophy, Metreleptin, Myalept, Myalepta, Novelion Therapeutics
0 Comment
News/ News/ R&D/ Rare diseases/ Transversal Categories
Shionogi’s Mulpleta gets FDA green light for patients with liver disease
Joy Persaud
FDA approval, lusutrombopag, Shionogi, thrombocytopenia
0 Comment
Market Access/ News/ News/ Rare diseases/ Top stories/ UK & Europe
Patients call for end to two-year row over CF drug funding
Richard Staines
cystic fibrosis, market access, NICE, Rare diseases, Vertex
0 Comment
Market Access/ News/ News/ News/ Oncology/ Rare diseases
Scotland’s NHS to fund Ipsen cancer diarrhoea drug
Richard Staines
cancer, Ipsen, market access, Rare diseases
0 Comment
Decision welcomed as step forward for patients