Labiotech.eu

Rare Disease Day: is a regulatory overhaul what the therapeutic field needs?

Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...

Rare Disease Day: Revolutionising clinical trial enrolment and retention with technology

Ruthie Davi, vice president of statistics and regulatory innovation at Medidata, highlights the value of technology in rare ...
Fierce Biotech

'Like talking to a brick wall': Senate hearing takes aim at FDA's rare disease review process

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the ...

Mother channels fear into advocacy after son’s rare disease diagnosis

Just days after her son was born, Julia Friar learned he had galactosemia, a rare genetic disorder that can be ...

IndoUSrare’s 2030 Vision to Transform Rare Disease Research: Diaspora Engagement, AI Innovation, Patient-Centered Action

Bridging Nations. Accelerating Therapies for Rare Diseases Together. The organization’s 2030 vision institutionalizes ...
STAT

FDA Commissioner Marty Makary defends rare disease drug rejections, Vinay Prasad

FDA Commissioner Marty Makary defended the agency’s recent rejections of rare disease drugs and one of his deputies, Vinay ...

FDA Commissioner Dr. Makary on rare disease therapy approvals, internal politics at the agency

FDA Commissioner Dr. Marty Makary joins 'Squawk Box' to discuss the agency's efforts to accelerate the development of ...

Podcast: Rare Disease Day: FDA guidance allowing advancement

Ahead of Rare Disease Day, Abigail Beaney, editor of Clinical Trials Arena, spoke with Robert Barrie, editor of Pharmaceutical Technology and Frankie Fattorini, reporter for Pharmaceutical Technology ...

Senate passes bipartisan resolution designating Feb. 28 as Rare Disease Day

The U.S. Senate unanimously approved a bipartisan resolution declaring Feb. 28, 2026, as Rare Disease Day, aiming to expand awareness, research, and support for millions of Americans living with rare ...
ABC 10News San Diego

Coronado teen loses sight at 13 to rare disease, finds new purpose running track

A rare genetic disease left a Coronado teenager legally blind at 13. Now he's running track — and helping drive research for ...

16-Year-Old Boy Diagnosed with Fatal Rare Disease Days After Brother Died from the Same Condition

"They had happy days on this earth," Azeza Kasham said of her late son, Hiatham, and her son, Gigi, both of whom were ...