Rare Diseases
Article
Osteogenesis Imperfecta Foundation Biennial Conference
Article
National Tay-Sachs & Allied Diseases Association Grants Available
Article
National Niemann-Pick Disease Foundation Seeks Executive Director
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HCU Network America Grant Requests
Article
Cornelia de Lange Syndrome Foundation Call for Abstracts
Article
Living With Aplastic Anemia Patient and Family Conferences
News
FDA grants breakthrough therapy designation for severe aplastic anemia drug
Eltrombopag, from Novartis, will get expedited review for first-line treatment of severe aplastic anemia. It is already approved as a second-line...
Feature
Gene silencer reduces mutant huntingtin protein in early-stage Huntington’s patients
Whether reducing the mutant huntingtin protein with the antisense oligonucleotide IONIS-HTTRx helps patients remains to be seen.