Top Medical News Today

Simon Ibell: Rare Disease Patient and Advocate (1979 – 2017)
Simon was a 4 ft 8 in motivational speaker and a passionate crusader for the rare disease community.
Morphsuits, Ed Sheeran, Prince Harry, and Batten Disease
Ollie, and his sister Amelia have Batten disease. Ollie's Army has helped the children meet Prince Harry and Ed Sheeran while also raising awareness of this rare disease.
Batten Disease: The Movie
Love Kennedy is about a young teenage girl named Kennedy Hansen, a Utah resident, who suddenly learns she has Batten disease.
FDA Approves Zykadia (ceritinib) As First Line Therapy for Rare Lung Cancer
Median PFS was 16.6 months in the Zykadia treated group compared to 8.3 months for the group given standard chemotherapy
GlycoMimetics' Drug Granted EU Orphan Drug Designation for AML Treatment
GlycoMimetics, Inc. announced that orphan designation for the company’s drug candidate GMI-1271 was granted for the treatment of acute myeloid leukemia (AML).
New Akcea Paper in NEJM Shows Favorable Cardiometabolic Effects with AKCEA-ANGPTL3-L Rx
Akcea Therapeutics published a paper titled "Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides" in The New England Journal of Medicine.
Cancer Crusader Loses Battle to Leiomyosarcoma
Amy J. Reed, MD, PhD, cancer crusader, passed away this week at the age of 44, in her home in Yardley, PA. She is survived by her husband and their 6 children.
Cannabidiol Reduces Convulsive-Seizure Frequency in Dravet Patients, Study Shows
Per NEJM, cannabidiol results in a greater reduction in convulsive-seizure frequency, and was associated with high rates of adverse events in Dravet patients.
Whipple's Disease Mimics Rheumatoid Arthritis, Causes Years of Improper Treatment
A study published in Orphanet Journal of Rare Disease indicates that the most common symptoms in Whipple’s disease mimic those found in rheumatoid arthritis.
Gene Therapy for Rare Blistering Skin Disorder Gets FDA Orphan Designation
Patients with EB have extremely delicate skin, often referred to as “butterfly skin,” that makes them extremely susceptible to blisters and poor healing of wounds.
RareDR Resource Guide
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