Entries by Chelsea's Hope

Update on the ION283 Clinical Pathway

/by

We understand many families are wondering what’s next for the development of the ION283 drug. Unfortunately, we received an update today from A.I.L.A. and Fondazione Telethon that Noventia Pharma returned the license for ION283 and will no longer be bringing it to a trial. Update on the ION283 Clinical Trial Pathway for Lafora Disease – […]

Announcing Lafora Therapy Town Hall

/by

  Questions about what therapies are next for the Lafora community? We are hosting a Lafora Therapy Town Hall to discuss what treatments the community can rally behind to bring from pre-clinical studies to clinical trials for our children. Join us next Tuesday, October 28th, at 11 am PDT via Zoom. As Lafora patient organizations – Chelsea’s […]

Invitation to Join Listening Sessions

/by

Families and caregivers, you might have seen in our July newsletter that we need your help to establish criteria for Centers of Excellence for treating Lafora disease. As an ultra-rare disease, most hospitals do not know what resources are needed in order to provide the best possible care for our children with Lafora. By establishing […]