Rare Disease Report


Top-line results from the pivotal Phase 3 GLOBE study of ofranergene obadenovec in combination with bevacizumab in patients with recurrent glioblastoma were reported today, and the study did not meet its primary endpoint of overall survival.
Monica Weldon, CEO, President and Founder of Bridge the Gap: SYNGAP Education and Research Foundation – one of Rare Disease Report’s Strategic Alliance Partners (SAP) – has made her debut as the lead author on a scientific paper.
Myasthenia gravis can be difficult to diagnose, and in this video, Beth Stein, M.D., Chief of Neurology at St. Joseph’s Regional Medical Center, discusses why the disease course makes it that way.
Ionis Pharmaceuticals reported positive top-line data from a completed Phase 1/2 study of RG6042 (Ionis HTTRx) in people with early stage Huntington’s disease .
In this video, Stephen Spaulding, M.A., Director Of Care Services for the ALS Association Greater Philadelphia Chapter, discusses how certain science and drugs can apply to more than one patient population.
George Yohrling, Ph.D., Senior Director of Mission and Science at the HDSA discusses the challenges that come with working with patients with HD, and communicating with their family members and caregivers.
Kazia Therapeutics has received orphan drug designation by the U.S. FDA for GDC-0084, intended for the treatment of glioblastoma multiforme.
AstraZeneca and Merck announced that selumetinib, its MEK 1/2 inhibitor for the treatment of NF1, was granted orphan drug designation by the U.S. FDA.
Data published in The Orphanet Journal of Rare Diseases confirmed that currently-available enzyme replacement therapies are unable to reverse or stabilize the progression of Pompe disease.
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