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 ...
The Press Democrat

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 ...
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 ...

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Today, the FDA issued draft guidance outlining a new regulatory pathway intended to help sponsors gain approval for highly ...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

Next frontiers of rare disease research

Scientific advances and cutting-edge technologies are reshaping how rare diseases are diagnosed and treated, bringing new hope to millions of patients and families. On Thursday, Feb. 24, Washington ...
The Barre-Montpelier Times Argus

AOP Health on Rare Diseases: Information. Collaboration. Innovation.

“Working in rare diseases since 1996 has shown us that innovation is never a solo effort,” emphasized Melissa Fellner, Vice ...
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 ...

FDA proposes framework to speed rare disease gene therapy approvals

The plan could provide patients earlier access to treatments that would otherwise stall because only limited data can be gathered.

Mstone Partners Healthcare Joins Global Efforts to Raise Awareness for Rare Disease Day

On the occasion of this Rare Disease Day, Mstone Partners Healthcare and our portfolio companies are proud to stand with the rare disease patient community, their families and caregivers, and ...

Tucsonans unite to raise awareness for Rare Disease Day

Families and advocates will gather Feb. 28 to spotlight rare diseases and connect those impacted in Southern Arizona.