Blog

Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.

December 16, 2025
Cure Rare Disease Awarded $7.4 Million CIRM Grant to Advance Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9

The program is designed with a novel, liver-detargeting, muscle-tropic capsid intended to improve safety and efficacy

Latest Perspectives

August 8, 2022
Cure Rare Disease Collaboration With Charles River Produces 3 Humanized Dystrophin Mouse Models
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August 1, 2022
JeanAnn Bryan
Caregiver to the Rare: What Goes Unseen & How to Help
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July 15, 2022
Michelle Werner
The Journey to Caffrey's Diagnosis
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July 11, 2022
Learn about disabilities through movies and TV shows
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July 7, 2022
Cure Rare Disease
Disability Pride Month
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July 1, 2022
AAV Neutralizing Antibodies  Frequently Asked Questions
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June 24, 2022
Fact Friday - LGMD2B
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June 17, 2022
Fact Friday - LGMD2G
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June 10, 2022
Fact Friday - LGMD2I
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June 9, 2022
What is CRISPR-mediated Exon Skipping?
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June 7, 2022
What are Antisense Oligonucleotides (ASOs)?
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June 6, 2022
Tips for Navigating College with a Rare Disease
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June 3, 2022
Fact Friday - ADSSL1-Gene Related Myopathy
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June 2, 2022
How can CRISPR and Gene Therapy Treat Duchenne?
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June 1, 2022
What is gene therapy?
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May 16, 2022
Eric Minghella
Social Security Disability Benefits with a Rare Disease
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May 13, 2022
Dana Edwards
Mental Health in Duchenne as a Caretaker
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May 11, 2022
How to Create a Facebook Fundraiser for Cure Rare Disease
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May 4, 2022
Sarah Krieger
Cure Rare Disease Lab Tour Day
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April 22, 2022
Accessible Spring Break Trips
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April 20, 2022
Casino Night 2022 Recap
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April 12, 2022
5 Easy Ways to Make Egg Hunts More Accessible
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March 28, 2022
Cure Rare Disease
Duchenne Myths and Misconceptions
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March 22, 2022
Cure Rare Disease
Let’s Talk: 5 Successful CRISPR Trials in Gene Therapy
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