FDA proposes new system for approving customized drugs and therapies for rare diseases

The FDA already authorizes the use of experimental drugs under what’s called “compassionate use,” for people with no other medical options. But the process is cumbersome to navigate and strictly ...

St. Louis Families Unite for Rare Disease Day 2026; Take Part Foundation Advances Critical Genetic Testing and Research

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and ...
BioSpace

FDA’s Bespoke Pathway To Focus on Gene Editing and RNA-Based Treatments for Rare Diseases

The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay ...
Healio

FDA provides new approval process for rare disease therapies without large clinical trials

The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based ...
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Today, the FDA issued draft guidance outlining a new regulatory pathway intended to help sponsors gain approval for highly ...
WYSO Public Radio

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 work — without requiring a clinical trial first.
Becker's Hospital Review

FDA unveils pathway for ultra-rare disease therapies

FDA unveils pathway ultra-rare disease therapies to approve gene-editing and RNA treatments when trials are unfeasible for small populations.

FDA proposes new treatment approval pathway for ultra-rare diseases

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

People with rare genetic conditions are ‘systematically ignored’ by NHS, report finds

Exclusive: One in four wait at least three years for diagnosis and many face treatment ‘access lottery’, according to report ...

Is Early Detection Of Rare Diseases Hidden In Your Genes?

In many countries, newborn screening programmes test babies for certain inherited conditions shortly after birth.

Rare disease pharmas could benefit from FDA guidance to accelerate development

FDA draft guidance could speed approval of individualized genome editing and RNA therapies for rare diseases. Read more here.