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Uncategorized

A total of 1961 posts are filed under Uncategorized
RARECast: Understanding Rare Disease Caregiver Needs
Rare disease caregivers are good at advocating for their family members, for research, and for the needs of rare disease patients broadly.… Continue Reading
Health Canada Kills Planned Orphan Drug Framework for Broader Review
Daniel S. Levine Durhane Wong-Rieger has long been working to get regulators in Canada to create an orphan drug designation similar to what… Continue Reading
RARECast: Screening for Rare Disease
The Rare Disease Film Festival will debut in Boston, MA on October 2-3, 2017. The festival will feature a range of more than 30 films… Continue Reading
FDA Awards Rare Pediatric Disease Designation to Sancilio Pharmaceuticals’ Sickle Cell Disease Therapy
Rare Daily Staff The U.S. Food and Drug Administration awarded Rare Pediatric Disease Designation to Sancilio Pharmaceuticals for the… Continue Reading
NIH awards nearly $100 million for Autism Centers of Excellence
Rare Daily Staff The National Institutes of Health said it made nine awards of research grants totaling nearly $100 million over the next… Continue Reading
Precision for Medicine Acquires Rare Disease-Focused CRO
Rare Daily Staff Precision for Medicine Acquires Rare Disease-Focused CRO Precision for Medicine said it acquired Agility Clinical, a… Continue Reading
Doctor, CSO, and Father: Hunting for Answers for Fragile X Children
Dr. Michael Tranfaglia serves as Medical Director and Chief Scientific Officer of FRAXA, a nonprofit organization based in Newburyport,… Continue Reading