News & Blog

Chelsea's Hope Lafora Children Research Fund Research Roundtable Thursday, April 27th, 10:30 am - 12:00 pm EST. Register to attend via Zoom We'll hear from Lafora Disease and Adult Polyglucosan Body Disease researchers around a common goal: reducing glycogen aggregation in the brain.

First Lafora Disease Research Roundtable

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A square image announcement for the Lafora Disease Research Roundtable. The top half of the image reads: Chelsea's Hope Lafora Children Research Fund Research Roundtable The bottom half contains the event details: Thursday, April 27th, 10:30 am - 12:00 pm EST. Register to attend via Zoom We'll hear from Lafora Disease and Adult Polyglucosan Body Disease researchers around a common goal: reducing glycogen aggregation in the brain. Please join us for our first Lafora Disease Research Roundtable!

When? It will be Thursday, April 27th, 10:30 am – 12:00 pm EST.

Where? The Roundtable will be virtual. You can register to attend via Zoom!

We’ll bring together Lafora Disease and Adult Polyglucosan Body Disease researchers around a common goal: reducing glycogen aggregation in the brain. The Research Roundtable will feature several presentations, followed by time for group discussion.

The presentations will be geared toward the science community as we work to build allies toward developing treatments for Lafora disease, but everyone is welcome. Families are welcome to attend and ask questions, so we hope you will be able to join us for this event!

It’s only one week away, so register today for the Lafora Disease Research Roundtable!

Also, please register if you are interested but you are unsure if you can attend or not! We hope to send the Zoom recording of the roundtable to everyone who signed up. However, we will not make the recording available publicly.

Chelsea’s Hope is excited for our first Research Roundtable. If successful, we plan to continue the series in the future. Please contact us before the roundtable if you have any questions.

Register for the Research Roundtable

Chelsea’s Hope Started a Volunteer Program

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We’re looking for the best kind of people: volunteers. Join a network dedicated to curing Lafora disease.

We’re excited to announce the launch of our volunteer program!! Please check out our new volunteer page and fill out the volunteer interest form to join the volunteer network.

You can make a difference with your skills and talents when you sign up to volunteer with Chelsea’s Hope! Partner with us to improve the lives of those affected by Lafora disease. You don’t need volunteer or work experience, but you do need passion and dedication.

Please spread the word that Chelsea’s Hope started a volunteer program! Share it with your friends and family, because we can do more together.
Volunteer with Chelsea’s Hope

Updated Patient Contact Form

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Families! Please complete our updated patient contact form! It is important to keep our community connected and our families informed.

Fill out the patient contact form

Update on Myozyme and Lumizyme drugs from Sanofi for Lafora Disease treatment

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Chelsea’s Hope continues to champion the development of effective Lafora disease (LD) therapeutics in both the research community and with interested companies. One recurring question is whether currently used therapeutics for other glycogen storage diseases could be used in LD. As you know, this would greatly benefit our community, allowing rapid therapeutic adoption.

Because of the similarities between LD and Pompe disease, there has been considerable interest if Myozyme and Lumizyme (from Sanofi Pharmaceuticals) could be effective in LD. The diseases are similar in the accumulation of aberrant glycogen, but different in where the aberrant glycogen is located. In LD, Lafora Bodies (LBs) are in the cytoplasm of cells, whereas in Pompe, the aberrant glycogen is in lysosomes. Because of the strong connections between Chelsea’s Hope and the LD research community, several groups have explored this possibility. Unfortunately, they have found that Myozyme does not effectively target brain LBs in LD mouse models. Given the similarities between Myozyme and Lumizyme, the results also strongly suggest that Lumizyme would be ineffective for LD. These data will be published in a peer-reviewed journal in the coming months. As a service to the community, Chelsea’s Hope wanted to be sure these data were communicated as soon as possible.

While disappointing, these results help to focus our efforts on the multiple therapeutic strategies which have shown to be effective in pre-clinical LD models. Multiple therapeutics were presented at the recent LD meeting, including cytoplasmic targeted enzymes and substrate reduction strategies. We continue to advocate for these to be developed and tested with maximal speed.