#RAREis Global Advocate Grant Awardee

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Read more here

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

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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!


Listen here

New Research on Gene Therapy and Lafora Body Aggregation in Mice

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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.

Check out the results of that study

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. 

Read Gene Therapy 101

New Research on Gene Therapy and Lafora Disease

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Researchers present gene replacement therapy as an approach for Lafora disease in the recently published article “Malin restoration as proof of concept for gene therapy for Lafora disease.” 

This paper shows that restoring malin expression in a mouse model that lacks it can reduce Lafora Body accumulation and neuroinflammation. 

This proof-of-concept suggests that it would be beneficial to develop gene therapy for restoring malin expression in patients where malin is mutated or missing.

If you have questions about gene therapy and Lafora Disease, please feel free to contact our Science Director, Dr. Kit Donohue, at katherine@chelseashope.org.  

Click here to read the publication