Top Medical News Today

Brain Cancer Patient to Run 7 Marathons on 7 Continents in 7 Days
47-year-old BethAnn Telford will run 7 marathons on 7 continents in 7 days. In 2005, she was diagnosed with brain cancer.
AHCF Fundraising, Awareness and Research Grants
In honor of our annual AHC Day, AHCF is excited to announce that we will be able to fund two research projects to get us one step closer to a treatment for AHC!
Tesaro Opens Expanded Access Program of PARP inhibitor for U.S. Ovarian Cancer Patients
Tesaro has opened up an expanded access program (EAP) for their PARP inhibtor, niraparib for ovarian cancer patients.
Subcutaneous Delivery of Daratumumab
Saad Usmani, MD, FACP, of the Levine Cancer Institute talks about the convenience and safety of subcutaneous delivery of daratumumab in multiple myeloma patients.
Rare Disease College Report: Nephropathic Cystinosis Scholarships Available
Global Genes and Horizon Pharma are partnering up to provide scholarships to nephropathic cystinosis patients for future schooling and career opportunities.
AHC Patients are Human Timebombs
Tomorrow, January 18th, is AHC day. AHC or alternating hemiplegia of childhood, is a devastating neurological disease caused by a mutation in the gene ATP1A3.
FDA Designates Cystic Fibrosis Lung Fungal Infection Treatment as QIDP
Pulmatrix's drug PUR1900 designed to treat fungal infections in the lungs of cystic fibrosis patients received Qualified Infectious Disease Product designation
Niemann-Pick Disease Type C Treatment Fast Tracked, Gearing Up for Trial
CTD Holding's treatment for Niemann-Pick Disease Type C (NPC) got Fast Tracked by the FDA and will go into a Phase 1 trial.
Fetal Alcohol Syndrome Surprisingly High Despite Educational Efforts
The study estimated that each year, 119,000 babies are born around the world with fetal alcohol syndrome.
NORD News for January - The Voice of the Community
The Voice of the Community: News from NORD and its Member Organizations – January 2017
RareDR Resource Guide
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