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Request for Proposals: The Marfan Foundation Research Grants
Schizophrenia’s Strongest Known Genetic Risk Deconstructed
Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find
CRISPR Helps Heal Mice with Muscular Dystrophy
Functional HIV Cure Step Closer To Reality With FDA Approval of Clinical Human Trials
Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Niemann-Pick Type C1 Disease
Rare Disease Day at NIH 2016
FDA Approves First Drug to Treat a Rare Enzyme Disorder in Pediatric and Adult Patients
GARD Information Navigator
Developing a New Tool to Detect a Frequently Missed Sex Chromosome Disorder in Boys
Have Questions About Genetic or Rare Diseases?
From the National Center for Advancing Translational Sciences
From the National Institutes of Health