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 ...

Transcript: Rare Diseases

Transcript: Rare Diseases ...
The Scientist

FDA Proposes Speedy “Plausible Mechanism Framework” for Rare Disease Drug Development

New FDA guidelines for personalized genomic drug development are a step forward in advancing new therapies for ultrarare diseases, but industry leaders need further clarification.
MedCity News

Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor

In the five years since Italy-based Chiesi Group established its rare disease division in Boston, the unit has landed regulatory approvals worldwide for 10 therapies — all small molecules and ...
Medscape

UK Regulator Targets Delays in Rare Disease Treatments

The Medicines and Healthcare products Regulatory Agency (MHRA) has announced plans to overhaul its approach to therapies for rare diseases. In a position paper, the agency highlighted that rare ...
Los Angeles Times

Contributor: By loosening standards, the FDA isn’t doing rare-disease patients any favors

This is read by an automated voice. Please report any issues or inconsistencies here. If you’re faced with a serious disease, you better hope it’s not a rare one. After an often tortuous path to ...
Hosted on MSN

Rare diseases affect 1 in 20 Indians: Doctor explains why awareness is the first cure

Rare diseases are medical conditions that individually affect a small proportion of people but when seen collectively, show a massive global health concern. In fact, 1 in 20 Indians are impacted by ...