The FDA has approved Rydapt for the treatment of adult patients with newly diagnosed acute myeloid leukemia in combination with chemotherapy.
Takeda Pharmaceutical and Harrington Discovery Institute announced that they have agreed to a partnership for rare disease treatment and therapeutics research.
Approval based on data from a Phase 1/2 study. BioMarin also received a Rare Pediatric Disease Priority Review Voucher which they can use on ANY future drug or sell it to another company.
Approved usaged of Stivarga (regorafenib) was expanded by the FDA and now includes treatment of patients with hepatocellular carcinoma (HCC or liver cancer).
A U.S. Senate panel voted to approve Dr. Scott Gottlieb as the head of the FDA. His nomination will be sent for a confirmation vote.
Rupesh Kumar, 21-year old of India, has been diagnosed with Progeria. He was once almost purchased and exhibited as a sideshow in the circus.
BridgeBio Pharma announced the launch of Eidos Therapeutics to develop an original small-molecule treatment named AG10 for transthyretin (TTR) amyloidosis.
Spinraza is indicated for “the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.” The definition, however, has not stopped some insurance companies from trying to limit which SMA patients receive the expensive drug – and at $125,000 per dose, the drug is expensive.
A systematic review of 58 studies involving Duchenne muscular dystrophy (DMD) by Leadley et al in the Orphanet Journal of Rare Disorders reaffirmed the tremendous cost this rare disease has on a family.