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Research

A total of 216 posts are filed under Research
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
Thanks to Rare Disease Report for this piece. University of Southern California (USC) start-up RASRx hopes to develop a drug to treat… Continue Reading
Castle Creek Announces First Patient Enrolled in DELIVERS Study of Diacerein 1% Ointment for Epidermolysis Bullosa Simplex
Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic… Continue Reading
Amicus Therapeutics Receives Rare Pediatric Disease Designation for SD-101 for Patients with Epidermolysis Bullosa
 Amicus Therapeutics, Inc.(link is external) (Nasdaq:FOLD), has received Rare Pediatric Disease designation from the U.S. Food and Drug… Continue Reading
IPF Patients Treated with Ofev® VS Placebo Were Twice as Likely to Have Improved or Stable Lung Function
Separate subgroup analysis demonstrates long-term efficacy of Ofev® in slowing disease progression is maintained for up to 96 weeks in IPF… Continue Reading
Parents of Children With Rare Disease Move Researchers to Connecticut
Story by Stephen Singer at The Hartford Courant Gayle Temkin spends her days at Solomon Schechter Day School, where she occupies a small,… Continue Reading
Abeona Therapeutics Announces New Updates on Sanfilippo Syndrome Clinical Trial
Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for… Continue Reading
National Leiomyosarcoma Foundation and Rare Cancer Research Foundation Team Up to Study Tumor Tissue
The National Leiomyosarcoma Foundation announces an exciting new collaborative partnership with the Rare Cancer Research Foundation (RCRF),… Continue Reading
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