European Medical Journal

Decoding Rare Immune Disorders: Interview with Filomeen Haerynck

My journey has been a long one. I am a paediatric immunologist and principal investigator of a PID research laboratory, and ...

Transcript: Rare diseases

Transcript: Rare Diseases ...
Morning Overview on MSN

DeepRare AI beats doctors in rare disease diagnosis test

A new artificial intelligence system called DeepRare has beaten experienced rare-disease physicians at their own specialty, ...

Rare disease isn’t rare—it’s a healthcare blind spot

In medicine, “rare” is often used to describe conditions that affect relatively few people. But when you work in healthcare ...
NPR

The FDA creates a quicker path for gene therapies

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new ...
Earth.com

New FDA framework could expand treatment access for rare genetic diseases

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...
JD Supra

Advancing Novel Treatments for Rare Diseases: FDA Unveils and Previews New Efforts to Assess Therapies for Very Small Patient Populations

Since taking the reins as FDA Commissioner, Dr. Martin Makary has repeatedly expressed an interest in new regulatory pathways, or new options for evidence generation, that would enable more flexible ...
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 ...
BioSpace

Rare Disease Groups Lobby for Regulatory Expediency as Makary Defends ‘Rigorous’ Approach

This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite ...
STAT

Don’t let hope expire for children with rare diseases

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...