FDA approves Duchenne muscular dystrophy treatment under ‘accelerated pathway’September 19, 2016Rare DiseasesNeurology
Case-control study points to Zika virus as cause of microcephalySeptember 15, 2016Rare DiseasesInfectious DiseasesZika
CANDLE syndrome case highlights key features of this type 1 interferonopathyAugust 27, 2016DermatologyRare Diseases
SHARE initiative releases consensus-based JDM management recommendationsAugust 26, 2016DermatologyMusculoskeletal DisordersRare Diseases
Study evaluates Zika syndrome with joint contracturesAugust 9, 2016Infectious DiseasesRare DiseasesZika
Children under 6 with factor XIII deficiency had no major bleeds with recombinant productJuly 28, 2016HematologyRare Diseases
Hemophilia drugs top Medicaid spending per prescriptionJuly 23, 2016HematologyRare DiseasesBusiness of Medicine
VIDEO: TNF inhibitors improved refractory skin disease in juvenile dermatomyositisJune 27, 2016DermatologyMusculoskeletal DisordersRare Diseases
Enlist appropriate psychological consults for gender dysphoriaJune 9, 2016EndocrinologyRare DiseasesMental Health
Materials help families find support for children with serious illnessesMay 24, 2016HematologyRare Diseases
Free Webinar Planned for Those Interested in Referring or Applying to NIH Undiagnosed Diseases Network May 13, 2016Rare Diseases
NORD to Develop Natural History Studies With 20 Rare Disease Patient OrganizationsMay 13, 2016Rare Diseases
