M2 Pharma - Therapy Areas - Hereditary Disorders

Therapy Areas: Hereditary Disorders

AMT's hemophilia B gene therapy gets FDA Orphan Drug status

4 January 2012 - Dutch human gene therapy specialist Amsterdam Molecular Therapeutics (AMS:AMT) said today its gene therapy programme for the treatment of hemophilia B had obtained US Food and Drug Administration (FDA) Orphan Drug status.

The company's hemophilia programme was granted ...

You must be logged in to view the whole article.
Click here if you require signing up

Related Headlines

Green Cross obtains approval in Korea for Hunter syndrome drug

Life Technologies forms JV with Chinese DaAn Gene for molecular diagnostic assays

Health Canada places Cinryze under Priority Review status

CSL Behring starts Phase II/III trial in haemophilia patients

Merck Serono launches validation study of PKU-QOL questionnaire

MDA grants Tivorsan USD1m to advance clinical work on DMD treatment

Prana gets FDA nod to initiate study of PBT2 in Huntington's disease

Biogen Idec, Isis form partnership for SMA antisense programme

Halo Therapeutics gets FDA Orphan status for HT-100 for DMD

AMT's hemophilia B gene therapy gets FDA Orphan Drug status

Synageva completes enrollment in Phase I/II study of SBC-102 for LAL Deficiency

Amicus Therapeutics completes enrolment in Phase III study of migalastat HCl

Selexys finalises Phase I trial for sickle cell compound

Baxter's ADVATE gets FDA approval for prophylaxis of Hemophilia A in adults, children

Michael Kelly joins Cure Duchenne as chief scientific officer