Key US senator investigates FDA over rare-disease drug denials

A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of ...
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 ...
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, ...
ScienceAlert on MSN

A Simple Photo of Your Hand Could Detect a Rare Health Disorder

(Kobe University) Palm reading may not foresee your future, but if you flip your hand around, it could reveal crucial information about how long you may live. According to new research, a simple photo ...
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 ...
The Tufts Daily

GUARDIAN: The newborn screening study redefining early detection for rare diseases

The survival rate in infants with severe combined immunodeficiency, a rare hereditary disease that prevents the body from ...

Mom noticed a faint discoloration on her 7-year-old’s skin — months later he was diagnosed with a rare disorder that slowly destroys skin

The first sign was so small it barely registered. A faint patch of discoloration on 7‑year‑old Lukas Caldwell’s cheek looked ...
The Coast News

Carlsbad family shares son’s battle with ultra-rare disease

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a ...

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 ...

As rare genetic disorder causes her son to regress, Carrboro mom teams up with UNC doctor studying new treatment

Hunter Syndrome is a rare genetic condition that can cause damage to the body and the brain. It often causes children to pass away in their teens, but a UNC Health ...
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 ...
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 ...