Larry Luxner,  —

Articles by Larry Luxner

Gene Therapy Takes Center Stage at 2019 NORD Summit

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

CureDuchenne Develops Biobank to Aid Duchenne Lab Research

CureDuchenne has begun taking blood samples and skin biopsies to facilitate research on new treatments for Duchenne muscular dystrophy. The CureDuchenne Biobank is a partnership involving neurologist Tahseen Mozaffar, MD, of the University of California-Irvine and RUCDR Infinite Biologics of Piscataway, New Jersey.

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…