Resources

Blog

Explore CRD's blog archive

Giving Tuesday 2024: Fuel Our Work

December 20, 2024
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Cure Rare Disease Expands Leadership Team and Board of Directors to Advance Mission

December 20, 2024
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Testing CRISPR Therapeutics in DMD Patient Cells: Process and Timeline

May 22, 2024
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Cure Rare Disease Celebrates the Grand Opening of New Laboratory

March 29, 2024
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Researcher Spotlight: Willeke van Roon-Mom

January 18, 2024
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SCA3 Program Overview

January 2, 2024
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Newsletters

Explore CRD's email newsletter archive

New from CRD: MyoTRIM Webinar & Upcoming Events

August 29, 2023

Our August newsletter is out. Save the date for our MyoTRIM webinar & check out an event near you.

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New from CRD: Patient Registry & More

June 29, 2023

Our June newsletter is out. Check out our Patient Registry, newest blogs and more.

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New from CRD: Rare Disease Day and More

February 28, 2023

February 2023 brings many exciting announcements from CRD, don't miss these updates!

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Publications and Whitepapers

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

Published November 4, 2024

Nature Reviews Drug Discovery

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Cure Rare Disease: An Initiative to Enable N of 1 Gene Editing

Published October 16, 2023

Human Gene Therapy

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Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Published September 27, 2023

The New England Journal of Medicine

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The Angel Treatment Pathway

In collaboration with reimbursement expert Eric Faulkner, former global VP of patient value and access at Novartis Gene Therapies, and the Muscular Dystrophy Association, we have developed an initial report to establish a novel pathway to advance treatments for ultra-rare diseases.

This is the first comprehensive approach mapped to date in the U.S. that offers a potential pathway for ultra-rare disease patients to access potentially life-saving therapies. The proposed pathway offers a precedent upon which we can rapidly build and execute this pathway in the U.S.

Complete the form to download the full text of the initial report.

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MyoTRIM - A Promising New Treatment for Muscular Dystrophy

Webinar

In this discussion with Dr. Noah Weisleder, PhD, from the Weisleder Lab at Ohio State University College of Medicine, and Rich Horgan, founder and CEO of Cure Rare Disease, we’ll delve into the promising research being conducted by Dr. Weisleders’s lab to develop MyoTRIM - a non-viral, protein-based therapy. We’ll discuss findings from the early-stage research, applications for the treatment of various forms of muscular dystrophy, and more.

Annual Reports

2023 Annual Report

2022 Annual Report

2021 Annual Report

2022 Form 990

Partner with us.

Your involvement helps shape our research and ensures our therapies meet real patient needs.

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