Recent FDA Orphan Drug Designations and Approvals Are Published
NORD has published a list of recent (over the past month) orphan drug designations and approvals.
China Establishes Its First Center Dedicated to Diagnosis for Undiagnosed Patients
In collaboration with NIH, China has established its first Undiagnosed Diseases Program center dedicated to helping solve rare disease medical mysteries. The center is based at Fudan University.
Alpha-1 Antitrypsin Deficiency Research Grant Is Available
The CHEST Foundation and Alpha-1 Foundation are sponsoring a one-year clinical research grant for study of alpha-1 antitrypsin deficiency. Applications are due April 30.
International Chordoma Research Workshop to Take Place July 14-15
The fifth International Chordoma Research Workshop will take place in Boston on July 14-15. Researchers from around the world will gather for this event, to focus on advancing the science and treatment of chordoma. Topics will include new insights into chordoma pathogenesis, novel therapeutic targets and upcoming clinical trials, among others.
National Research Conference Planned for Rett Syndrome and Related Diseases
A landmark national research symposium will take place near Chicago June 21-24 for Rett syndrome, CDKL5 disorder, FOXG1 disorder and MECP2 duplication. This first-ever collaboration will be followed by a family conference June 24-26.
Clinical Practice Guidelines for Preschoolers With Cystic Fibrosis Are Published
A multidisciplinary committee convened by the Cystic Fibrosis Foundation has developed and published comprehensive evidence-based and consensus recommendations for the care of preschool children with cystic fibrosis.