News & Blog

New article about rare disease community

Though we face different fights, rare disease families take similar journeys to save their children. Other families affected by rare diseases like Lafora are starting companies to help develop treatments for their children. We are grateful to partner with Vibe Bio in our efforts to find and fund a cure for Lafora disease. 

#RAREis Global Advocate Grant Awardee

We’re excited to announce that we’ve been selected as a #RAREis Global Advocate Grant awardee! We look forward to continuing to make a positive impact in our rare disease community by using this grant to increase education and awareness around Lafora Disease.  

We’re proud to be selected and look forward to expanding our education and awareness programs. We’re grateful to Horizon Therapeutics for their recognition of our efforts to support the Lafora Disease community.

Clubhouse Podcast

Listen to Alok Tayi and Joshua Forman discuss their company Vibe Bio and their fight against rare disease on the Gene Fixers podcast.

We are glad to partner with Vibe Bio!

New Research on Gene Therapy and Lafora Body Aggregation in Mice

Dr. Berge Minassian’s lab has developed and tested an AAV gene therapy for Lafora Disease to reduce glycogen synthesis and halt Lafora Body aggregation in mice.

We are still a long way from being able to use gene therapy to treat Lafora Disease, however, this is an important step toward curing it for future generations.

If you have questions about gene therapy and Lafora Disease, check out our easy-to-read guide linked below. Please feel free to contact our Science Director, Dr. Kit Donohue, at katherine@chelseashope.org