News

Capitol with RARE Hope logo
News, Press Release, Advocacy News

RARE Hope Marks Bipartisan Introduction of the Genomic Answers for Children’s Health Act with Partner Organizations

Nina Frost presenting at NIH Science Meeting
Event

RARE Hope + NIH Science Meeting Brings Global Experts Together to Advance Shared Pathways in Neurological Disease

Photo-©-Tony-Powell.-A-Brilliant-Night-for-Rare-Hope.-National-Cathedral.-November-13,-2025-3.jpg
Fundraising

A Brilliant Night for RARE Hope Fills the National Cathedral With Inspiration, Generosity, and Breakthrough Momentum

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

RARE_Hope_A_Brilliant_Night_Background_Graphic.jpg
News, Fundraising

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

Pediatric_Rare_Disease.jpg
Press Release

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

DavidLiu_CaseyAtkins_1_mo-BROAD00254719.jpg
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.