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 ...
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.

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 ...

DeepRare AI outperforms doctors on rare disease diagnosis in head-to-head test

Rare diseases are complex medical disorders that are notoriously difficult to diagnose because many present with a wide variety of symptoms that can overlap with more common illnesses. Currently, ...
Rutland Herald

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 ...

Jesse Jackson’s Rare Brain Disease: Neurologists Explain PSP

Attention has turned to progressive supranuclear palsy, a rare neurodegenerative disorder. Here's what it is and why it's ...