Ara and Bethany, Ara's mom, live in Newton, Alabama, and Ara has the rare disease. A Wiregrass family is battling MPS 1, a form of the rare genetic disease. To help fund Ara's transplant, visit the family's goFundMe at https://www.gofundme.com/3qqoe5sFor more information about MPS 1 and MPS, visit mpssociety.org Bethany, Ara's mom offered some words of advice to those who are dealing with MPS 1. "You have to have a good team with you," said Bethany Hampton, Ara's mom.
Ionis says it gathered promising PhIII data for a top rare disease drug, but serious safety questions remain – ENDPOINTS NEWS
Its lead late-stage drug patisiran could prove a safer alternative to Ionis' drug, which is why the Cambridge-based biotech's facebook/" target="_blank">shares shot up 15% this morning. Researchers said that there were three serious cases of thrombocytopenia, or low platelet levels, that were triggered by the drug. The FDA once put this drug on hold, causing GlaxoSmithKline to edge away from their collaboration on the drug. When the safety issue first came up last year, triggering the hold, GSK opted to shelve plans for a Phase III study of TTR amyloid cardiomyopathy. Ionis Pharmaceuticals $IONS says that its Phase III study of inotersen (IONIS-TTRRx) hit both primary endpoints in patients with rare cases of familial amyloid polyneuropathy (FAP).collected by :Lucy William
No comments:
Post a Comment