19 June 2018 - Submission in the European Union on track for mid-year and in Japan for the second half of ...
6 June 2018 - Cellectar Biosciences announces today that the U.S. FDA has granted rare paediatric disease designation to CLR 131, ...
24 May 2018 - Krystal Biotech today announces that the U.S. FDA has granted fast track designation to KB103 for ...
24 May 2018 - First enzyme therapy to treat phenylketonuria. ...
2 May 2018 - Phase I/II clinical trial expected to enrol children with MPS II. ...
25 April 2018 - Phase 1/2a clinical study of SYNB1618 evaluating safety and tolerability as well as exploratory endpoints expected ...
18 April 2018 - Taking the rebates that it expects to negotiate into consideration, Ultragenyx said that burosumab will cost approximately ...
19 March 2018 - FDA statisticians pondered changing how the agency uses statistics to approve drugs for rare disease at ...
26 February 2018 - Today 30 million people in the United States – or one out of every 10 Americans – ...
22 February 2018 - Genomic characterisation of cancers has shown that some oncogenic alterations occur at very low frequency and are ...
20 February 2018 - PTC Therapeutics today announced that the Office of New Drugs of the U.S. FDA has reiterated ...
12 February 2018 - Symdeko to begin shipping to pharmacies this week. ...
12 February 2018 - Six month PDUFA goal date is 13 August 2018. ...
5 February 2018 - CAP-1002 to benefit from expedited review program for drugs for unmet needs. ...
1 February 2018 - PDUFA date set for 11 August 2018. ...