The study found the drug to be safe and clinical remission was achieved in the majority of patients.
For quite some time, the only treatment option for people with hereditary tyrosinemia type 1 (HT-1) was SOBI’s Orfadin (nitisinone). That is changing.
Horizon Pharma's shares are down due to their Phase 3 trial for the treatment of Friedreich’s ataxia not meeting its endpoint.
According to Jenkins, he has been thinking about leaving the FDA since 2015 but has stayed on to make sure that a person was in place to assume his responsibilities.
RDR has compiled a few awesome photos from Facebook about the Cures Act and #CuresNow. Vote for your favorite photo!
RDR presents a live Twitter feed of people talking about #CuresNow.
Shruti Chateurvedi and colleagues looked at the proportion of patients with thrombotic thrombocytopenic purpura who reported symptoms of depression and post-traumatic stress disorder (PTSD).
A new study published in the New England Journal of Medicine questions the ability of whole genome sequencing to be the best means to diagnose patients.
The vote by the Senate was 94 – 5. Last week, Congress voted 392 – 26 in favor of the Bill.
Ruben Mesa, MD of the Mayo Clinic in Scottsdale Arizona talks about two approaches to treating polycythemia vera, and essential thrombocythemia that use interferons.