Go behind the scenes of this year’s Drug Development Forum When Dan Perrien attended his first FOP Drug Development Forum (DDF), he met two young children with fibrodysplasia ossificans progressiva (FOP). He watched them run and play like any other children would — while the adults in the room were on high alert to make […]
Meet 12-year-old Raina When Raina and her friends created a club called “The Underpuppies,” they decided on just one requirement: You have to be genuinely kind to join. To enforce that requirement, they developed a test. The current Underpuppies stage a situation where the prospective member has to help someone in need or be willing […]
Blueprint Medicines Corporation, a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, today announced preclinical proof-of-concept data for BLU-782, an investigational oral precision therapy specifically designed to target mutant activin-like kinase 2, the underlying cause of fibrodysplasia ossificans progressiva. Preclinical studies in a well-characterized, genetically accurate FOP model showed […]
Clementia Pharmaceuticals Inc., a clinical-stage biopharmaceutical company innovating treatments for people with ultra-rare bone disorders and other diseases, today announced updated data from the open label extension (“Part B”) of its ongoing Phase 2 clinical trial of palovarotene in fibrodysplasia ossificans progressiva (FOP). “These updated data continue to support the potential for palovarotene in FOP, […]
Human skeletal stem cells that become bone, cartilage, or stroma cells have been isolated from fetal and adult bones. This is the first time that skeletal stem cells, which had been observed in rodent models, have been identified in humans. The researchers were also able to derive the skeletal stem cells from human induced pluripotent […]
Learn more about Regeneron’s LUMINA-1 clinical trial presented by Dr. Richard Keen from the Royal National Orthopaedic Hospital in England. This webinar is hosted by FOP Friends in the United Kingdom http://fopfriends.com/. Source: FOPnews
