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 ...
Fierce Pharma

CDMO Quotient extends Ipsen supply pact for rare disease drug Sohonos

Quotient Sciences, a CDMO that specializes in "niche and smaller volume" drugs that it says are often overlooked by bigger contract manufacturers, has ...
Fierce Biotech

FDA details rationale for rejecting rare disease gene therapy from Regenxbio

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...
Medical Marketing & Media

5 outstanding rare disease campaigns to spotlight Rare Disease Month

This article is part of a Rare Disease series sponsored by DeepIntent. With Rare Disease Month wrapping up and Rare Disease Day taking place on Saturday, MM+M is celebrating a handful of campaigns ...
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 ...

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 ...

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 ...
WISH-TV

Pastor’s voice lost from rare disease, restored with FDA-approved treatment

Recurrent respiratory papillomatosis (RRP) treatment finally approved by FDA, targeting the root cause of the disease and ...

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 ...
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 raises awareness for patients, families in Chicago area

Today is Rare Disease Day and it's meant to raise awareness for patients and families dealing with rare dieseases.

Colorado family pushes for change after rare disease clinical trial abruptly ends

A Colorado family is calling for change and sharing their struggle after a clinical trial their son relied on suddenly ended.