The American Journal of Managed Care

FDA Proposes New Approval Pathway for Individualized Ultra-Rare Disease Therapies

Today, the FDA issued draft guidance outlining a new regulatory pathway intended to help sponsors gain approval for highly ...
University of California

From Rett and Pitt-Hopkins to ALS: 5 rare diseases UC San Diego researchers are tackling in the lab

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...

FDA proposes new treatment approval pathway for ultrarare diseases

The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments ...
PharmTech

Impacts of FDA's Plausible Mechanism Framework for Ultra-Rare Disease Therapies

FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.
TheHealthSite

Rare Disease Day 2026: Experts Reveal Why Certain Disorders Target Brain And Nervous System

Rare disease day is observed annually on the last day of February to spread awareness on certain conditions that can impact on patients lives.

A New FDA Plan Could Transform Treatment Access for Rare Disease Patients

The framework was released as draft guidance on February 23.
Opinion
TownhallOpinion

FDA Cruelly Holding Up Approval of Treatments for Rare Diseases, Despite Children Likely to Die Soon

The U.S. Senate Special Committee on Aging conducted a hearing on February 26 to investigate why the FDA is holding up the approval of desperately needed new drugs for those with rare diseases. Many ...
pharmaphorum

FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its new regulatory pathway to market for individualised therapies that treat ultra-rare diseases, based on a 'plausible mechanism' premise.
TheHealthSite

Rare Neurological Disorders: Subtle Early Brain Symptoms You Should Never Ignore

Unusual headaches, vision changes, mood shifts or numbness? Know the subtle early warning signs of rare neurological ...
KLCC

The FDA creates a quicker path for gene therapies

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...
Medical Xpress

Stone baby: The rare condition that produces a calcified fetus

For some women, pregnancy is a time of profound loss. Not all pregnancies progress as expected. One serious complication is ectopic pregnancy, a condition in which a fertilized egg implants somewhere ...