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:
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.
Frank Harris, Ph.D.
President – Chelsea’s Hope Lafora Children Research Fund
Chelsea’s Hope Hosts Research Webinar
On June 7, 2021, Chelsea’s Hope was thrilled to be able to host a research webinar on Lafora Disease. We were joined by Matt Gentry of the University of Kentucky College of Medicine and the Lafora Epilepsy Cure Initiative (LECI) and Dustin Armstrong and Rob Shaffer of EnAble Therapeutics joined us to discuss the current state of LECI research and exciting new research being done with EnAble Therapeutics.
Chelsea’s Hope thanks Drs. Gentry, Armstrong, and Shaffer for meeting with our families via this webinar. Additional thanks are due to Lena Ismail and Sheila Barter for arranging this event.
A recording of the webinar can be viewed here.
Due to continued COVID restrictions, the July 2021 LD meeting has been rescheduled for September 20th – 21st, 2021 at the Sanford Consortium for Regenerative Medicine in La Jolla, CA. The meeting will be both in person and available on-line. Presentations will begin in the early afternoon on September 20th with an update regarding current research and efforts to move therapies into the clinic. This update will be followed by scientific talks, time for Q&A, and the evening will conclude with a meal to promote discussion and interactions. September 21st will be a full day of scientific talks focused on LD research with a poster session and meals provided.
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.
Take a survey to help better understand what it’s like living with Lafora disease (LD) and to help guide development of new potential LD therapies.
Learn more here: Calling All People and Caregivers
Just Like A Butterfly by Niki Markou
“Just Like A Butterfly” Audio Release Date: February 10th, 2021, available on multiple digital music platforms like Spotify, Apple Music & TikTok. We ask you stream the song and also make a donation! Help save our children.
“This journey has been extremely difficult and devastating and we do not want to lose our beautiful girl. I have written a song that is about how she bounces back from her seizures and what I wish for her ” – Mother, Niki Markou
Dear Lafora Families,
With funding from the Global Genes’ Continuity of Care RARE Patient Impact Grant, Chelsea’s Hope is offering grants to Lafora Households Impacted by COVID-19.
Limited funds are available, but we hope to help as many patients as possible. The 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 form.