Increased red cell distribution width in Fanconi anemia: a novel marker of stress erythropoiesis
25 July 2016
Breakthrough status for Pfizer/Spark haemophilia gene therapy
22 July 2016
EU orphan status for Cell Medica drug
21 July 2016
HoFH Drug Juxtapid No Longer Available in EU
21 July 2016

Aegerion has announced they will no longer market Juxtapid (lomitapide) in Europe for th

Celgene, Jounce in $2.6-bn immuno-oncology deal
20 July 2016
Combining Immune and Gene Therapy For 2 Rare Diseases
20 July 2016

Selecta Biosciences is assisting in the development of gene therapies for Methylmalonic

FDA Approves HAE Drug Berinert For Pediatric Use
19 July 2016

The FDA has  read more...

Narcolepsy Drug Gets FDA Orphan Designation
12 July 2016

The FDA has granted an Orphan Drug Designation for Mazindol, to treat Narcolepsy.