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

New Research on Gene Therapy and Lafora Disease

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  

Gene Therapy 101


Questions about gene therapy and Lafora disease? To access the easy-to-read guide Gene Therapy 101:

If you want to read more about gene therapy, this is a good patient-friendly website.

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


Patient Community Meet & Greet with New Science Director

Please join our new Science Director, Dr. Kit Donohue, for a community meet and greet. As Kit steps into her new role, she would like to hear from you – the patient community!

Kit has been fortunate to study in Dr. Matthew Gentry’s lab for the past five years, where she met and collaborated with many of the scientists seeking treatment for Lafora Disease. The progress that has already been made is a testament to what can happen when scientists work closely with the patient community.

Kit is excited to play a role in deepening that collaboration as we move closer toward the goal of finding a cure for Lafora Disease. We hope you will join and share your needs, questions, concerns, and hope for the future of our community.

When: June 29, 2022 06:00 PM in Eastern Time US and Canada. Registration before June 29, 2022 is required.

If you are unable to attend, would like to submit a question/thought ahead of time or privately, please email

Vibe Bio Launches To Transform Drug Development For Patients With Rare Diseases

More information on new biotech company Vibe Bio and founder Alok Tayi helping Chelsea’s Hope and patients with rare disease by using crypto to support the development of treatments.

To read article