EnAble Therapeutics Expanded Access Announcement

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EnAble Therapeutics has informed Chelsea’s Hope that it is willing to partner with clinicians to lead a n=1 Expanded Access trial or through the Right to Try Act with their potential therapy for Lafora Disease, VAL-1221.

EnAble has indicated that the following medical resources and support will be needed:
Clinicians with institutional support including internal and external regulatory assistance and treatment / I.V. infusion capability.

EnAble has confirmed that this could be your child’s primary neurologist but EnAble has also confirmed that the clinician does not need to be a neurologist.

If interested, please have your clinician contact:

Vicki Wong
Enable Therapeutics
vwong.nobleterra@gmail.com

2021 Lafora Symposium Delayed Due to Continuing Pandemic

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To Chelsea’s Hope Families, Researchers and Clinicians,

I regret having to inform you that we must delay the 2021 Lafora Symposium. While international health conditions are improved over last year, the impact of the pandemic continues. The University of California at San Diego, our venue for the symposium, is unable to host outside groups due to the pandemic. Additionally, most of our researchers and clinicians from outside the United States, as well as some of our domestic researchers are unable to travel for the symposium.

While we have to delay this year’s symposium, I want you all to know that the work continues. Our researchers, who have made so many significant advances, continue to collaborate to bring forth a cure for Lafora. Chelsea’s Hope continues to have regular meetings with Dr. Matt Gentry and other members of the Lafora Epilepsy Cure Initiative (LECI), as well as representatives from Ionis and EnAble and we are always looking for opportunities for our families to meet with our researchers. Additionally, Dr. Gentry is leading the effort on behalf of the LECI to apply for renewal of the NIH PO1 research grant.

Chelsea’s Hope is working with the LECI to reschedule the symposium. We will report the reschedule date as soon as possible.

With Hope,

Frank Harris, Ph.D.
President – Chelsea’s Hope Lafora Children Research Fund
fharris@chelseashope.org

Researchers Have Discovered the Cause of Neurodegeneration in Lafora Patients

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One of the more horrifying impacts of Lafora Disease is the neurodegeneration that these children suffer. Families bear witness as their children suffer in frustration from their own cognitive decline. Researchers now understand the cause of this degeneration. As Dr. Jordi Duran explains in this article, “For years it was believed that the disease was caused by the accumulation of Lafora bodies only in neurons, but now we have shown that neurodegeneration is caused by accumulations in glial cells.”

Chelsea’s Hope is thrilled to thank Dr. Guinovart, Dr. Duran, and their entire staff of researchers for this great discovery. We also offer our sincere thanks to Dr. Matt Gentry and Dr. Jose Antonio del Rio for their important support and contributions to this discovery.

Chelsea’s Hope and all the Lafora families desperately hope for a cure for Lafora. Drs. Guinovart and Duran have gotten us a step closer.

Grant for Lafora Households: Chelsea’s Hope Patient Assistance Deadline Extended

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***GRANT FOR LAFORA HOUSEHOLDS AWARDED; FORM CLOSED***

Dear Lafora Families,

With funding from the Global Genes’ Continuity of Care RARE Patient Impact Grant, Chelsea’s Hope is offering grants to Lafora Disease Households impacted by COVID-19.

Limited funds are available, but we hope to help as many patients as possible. The grant application deadline has now been extended a week to December 7, 2020. All applications will be reviewed with the goal of making awards by December 15, 2020. Please contact Chelsea’s Hope with any questions or for help completing the grant application form.

Chelsea’s Hope Patient Assistance Grant