rare disease
Celebrations for ASMD community as EU clears Sanofi’s Xenpozyme
Phil Taylor
EU, Genzyme, rare disease, regulatory approval, Sanofi, Xenpozyme
0 Comment
Data backs AZ’s Wilson disease drug, but with a question mark
Phil Taylor
Alexion, AstraZeneca, rare disease, Wilson disease
0 Comment
Sarepta shares yo-yo as FDA places hold on Duchenne trial
Phil Taylor
Duchenne muscular dystrophy, Exondys 51, fda, rare disease, regulatory, safety, Sarepta, vesleteplirsen
0 Comment
AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen
Phil Taylor
antisense oligonucleotides, AstraZeneca, ATTR amyloidosis, Ionis Pharmaceuticals, rare disease
0 Comment
Second unanimous FDA adcomm vote boosts bluebird bio
Phil Taylor
beta thalassaemia, bluebird bio, fda, gene therapy, rare disease, regulatory
0 Comment
FDA panel backs bluebird’s CALD gene therapy, despite safety worries
Phil Taylor
bluebird bio, CALD, fda, gene therapy, rare disease, regulatory
0 Comment
Market Access/ News/ News/ News/ Patients
UK grants early access to Argenx’ efgartigimod for myasthenia gravis
Phil Taylor
access, Argenx, efgartigimod, MHRA, myasthenia gravis, patients, rare disease, UK, Vyvgart
0 Comment
News/ News/ Sales and Marketing
FDA grants speedy review to Dupixent rare skin disease
Phil Taylor
dermatology, Dupixent, prurigo nodularis, rare disease, Regeneron, regulatory, Sanofi
0 Comment