News

Family with images of DNA

News Research

Family enlists Harvard scientists in fight against brain disease

Girl paiting with her parents

News Research

Turning rare disease research into a common goal

2025-07-17 Liu Lutz image

Research

First-in-Neuro Breakthrough: Next-Generation Genetic Editing Tool Rescues Devastating Childhood Brain Disease in vivo

2025-05-14 Iceland Photo for Website.jpg

News Research

RARE Hope Leads Global Scientific Working Session in Iceland to Advance Therapeutic Strategies for AHC

News Fundraising

Announcing A Brilliant Night for RARE Hope, a Gala Fundraiser Event

Press Release

RARE Hope Joins DATAcc by DiMe to Accelerate Development of Digital Measures for Pediatric Rare Disease

Research

David Liu receives Breakthrough Prize in Life Sciences

The Jackson Laboratory and Unravel Bioscience logos

Research

Unravel Biosciences and The Jackson Laboratory announce collaboration

Simon Frost and his daughter Annabel

Advocacy News

American man's fight to save a girl in Gaza he's never met

The Abu Zaiter Family

Advocacy News

American families working to evacuate Gaza child facing rare medical disorder

Annabel Frost and Julia Abu Zaiter

Advocacy News

In Washington DC and Gaza two very different families are united by one very rare disease

Cat Lutz working with her team at a bench in a laboratory

Research

The Jackson Laboratory receives $8 million in federal funding to expand research capacity in rare diseases

Images of Nina Frost, Mary Saladino and Dr. Cat Lutz

Research

Patient Partners: Harnessing synergies to pursue ASOs as part of the AHC research portfolio

Simon Frost

Research

His daughter Annabel has a rare disorder. He’s developing a novel gene therapy.