The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora Disease and help accelerate the development of treatments.
Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 non-profit organization. EIN: 27-1008382
Chelsea’s Hope, Post Office Box 348626, Sacramento, CA 95834
Lafora Body Disease Day 2024
/by Christine KellyThis year’s Lafora Body Disease Day holds special significance. Not only are we in the middle of our first Awareness Campaign, but today would have been Chelsea’s 34th birthday – our namesake and the inspiration for Chelsea’s Hope Lafora Children Research Fund. She, along with all the children we have lost to Lafora disease, is the reason we continue the fight. Keep reading for a message from our founder!
Share How You #FightLafora
We seek to honor all our Lafora warriors and heroes as we continue along the therapeutic journey.
Help us reach a cure by joining in! Here’s how you can raise awareness and fund the ION283 Safety Study for Lafora disease:
Creating awareness helps identify more patients, find partners, and expand our community dedicated to reaching treatments for Lafora disease. So many people don’t know Lafora disease exists. Together, we can change that!
Download and share a graphic:
Let’s honor those we’ve lost and continue to fight for our children facing Lafora disease today. Thank you for your support in raising awareness and funding the Safety Study.
A Note from Linda
As many know, I am the founder of Chelsea’s Hope Lafora Children Research Fund, but my most important role and privilege was being Chelsea’s mom.
October 1st, also deemed Lafora Awareness Day, is Chelsea’s birthday; she would have been 34 years old.
She left this world much too soon, in December 2016, at the age of 26.
I began Chelsea’s Hope 15 years ago as a means to connect Lafora families for support, raise awareness, and fund research for a cure. We have succeeded on these points, and I am so proud to share that this past week, the FDA approved the first-ever patient safety study. This is HUGE news!
Please join me in helping fund this historic and life-changing moment in Chelsea’s Hope history. The families of Chelsea’s Hope are desperate to save their children’s lives.
So many of you have donated to our research pleas over the years, and we are forever grateful! Your generosity has made a huge difference to our success. We have now reached this pivotal juncture of clinical trials and are on the cusp of treatment that could positively change the course of life for Lafora patients.
Please donate, join our team, and/or share our campaign to make history by eradicating Lafora disease and saving the lives of our children!
With gratitude and always maintaining HOPE,
Linda
You can still join the Awareness Campaign as a fundraiser (here’s a video to get you started!) or give to support the Safety Study. Chelsea’s Hope also prepared a toolkit to help and will update our Awareness folder with more graphics you can share.
ION283 Safety Study on ClincialTrials.Gov
/by Christine KellyA Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease – ClinicalTrials.gov
We are thrilled to announce that the ION283 Safety Study information was posted to clinicaltrials.gov. Read the full inclusion and exclusion criteria HERE.
Please share the protocol with your clinicians to determine if your loved one is eligible. The essential eligibility criteria is:
At this time, the study is not enrolling patients. However, they have posted the details for families to prepare. If you or your neurologist contact the UTSW team through the clinicaltrials.gov website, you will receive an email instructing you to contact your country’s patient organization for an application packet that your neurologist must complete. However, the patient organizations have not yet received this application packet. Dr. Minassian and his team are waiting for approval of the application packet by the university. They think the packet will be approved sometime in October.
How will the enrollment process work?
When the packet of information is released, neurologists must complete the information requested, and email the packet to the team at UTSW (that contact email will be provided in the packet). The team at UTSW will keep a record of the order in which the packets arrive based on the timestamp of the email, and they will use that list to accept patients on a first-come, first-serve basis.
Patients will not know what number they are on the list. If they receive a spot in the study, they will be contacted when there are enough funds to enroll them.
We will continue to update our patient community as new information becomes available. If you have questions about the application process, contact our Science Director, Dr. Kit Donohue.
Noventia Statement for the Lafora Community
/by Christine KellyDear Lafora Community,
A few weeks ago we finalized an agreement for the rights to ION283, Ionis’ investigational medicine for the treatment of Lafora disease.
Since its founding, Noventia Pharma, a privately owned European specialty pharmaceutical company, has been committed to advancing research to bring hope to people living with rare diseases, particularly those affecting the CNS.
Noventia will now assume full responsibility for the development of ION283 and we are excited to apply our know-how, expertise, and passion to this project.
Over the coming weeks, Ionis will transfer all materials and data related to ION283 to Noventia. Noventia, together with other stakeholders, will then determine the feasibility of clinical development and the next steps for the program.
Our current objective is to start clinical trials as soon as possible and generate data to evaluate ION283 for potential registration in Europe and the US.
For this reason, Noventia is setting up an International Advisory Board to guide and support our efforts. We aim to work in partnership with the European Medicines Agency, the Food and Drug Administration, and Patient Associations where possible to speed up all these processes. We expect the recently initiated clinical trial at UT Southwestern to continue during this time.
We are grateful for the support, collaboration, and perseverance of the Lafora community. We look forward to working with you to expedite this transition and facilitate the continuation of development activities for ION283.
Sincerely,
Massimo
Massimo Radaelli
President & CEO
Noventia Pharma
Chelsea’s Hope Joins FACE It 2024
/by Christine KellyWednesday, September 18th, is Childhood Dementia Day, and we’ve joined the FACE It awareness push to make childhood dementia impossible to ignore. 2024 is the second year Chelsea’s Hope is participating in the movement. We invite you to join us!
The Childhood Dementia Initiative (CDI) started the FACE It campaign to raise awareness of childhood dementia, which so many people don’t know exists.
What is childhood dementia?
We see what dementia does to our loved ones with Lafora disease. Most people associate the life-altering symptoms of dementia with older people, but according to our friends at CDI, more than 100 genetic conditions cause dementia in childhood. The prognosis for children with childhood dementia is dire: 50% die before the age of 10, and 70% before reaching adulthood. All will die prematurely. View a childhood dementia fact sheet HERE.
On September 18th, we invite you to make your face impossible to ignore! Join the fun with face paint, makeup, or a filter before posting to social media. Together, we’ll draw attention to childhood dementia and fight Lafora disease.
Be sure to send Chelsea’s Hope your photos or tag us if you want us to feature you on our social media. Use the hashtags #ChildhoodDementia and #FACEit to join. You can also check out CDI’s FACE it kit with tips and resources to raise awareness.
So, will you join the movement?
Ionis Statement for the Lafora Community
/by Christine KellyThe licensing of the ION283 program will not impact the Safety Study. That will continue as planned, and we will update our community as soon as enrollment is open on clinicaltrials.gov.
As a reminder, please sign up for our mailing list and indicate you want to receive “research updates” to receive this news as soon as possible.
ION283 Safety Study Approved
/by Christine KellyWe just learned from Dr. Minassian that the FDA has approved the ION283 Safety Study!
We are so grateful for your continuous support, for Dr. Minassian and his team at UTSW, Dr. Messahel, IONIS, and everyone involved for getting us here.
Our patient community has been a driving force in supporting reaching this stage and we can celebrate another step forward today. We hope this will lead to treatments for our children.
We do not yet have the application site on clinicaltrials.gov or the criteria for inclusion in the Safety Study. Those will be coming in a few weeks. In the meantime, we can continue fundraising and ensuring all patient families learn of this opportunity.
Budget
The estimated cost of the Safety Study is $1.5 million USD. Please remember, donations do not guarantee a spot for your loved one in the trial. We encourage donations through a patient organization. Chelsea’s Hope has an online fundraiser where you can donate and we will transfer the money to UTSW.
France Lafora is also fundraising HERE.
Please help us share this information!
If you did not get an email update from us earlier, please ensure you are signed up for our mailing list. Once we learn the criteria from Dr. Minassian, we will send out the information to everyone who has signed up for “research updates.” You can contact christine@chelseashope.org to confirm your email preferences with the subject “Check my Chelsea’s Hope Email Preferences.”
Questions? Please check out our FAQs section on our webpage.
We answer commonly asked questions about the safety study, including what it is, how much it costs, who can participate, and more! If you don’t see an answer to your question, please email katherine@chelseashope.org with the subject “Safety Study Question.”
Announcing a 2024 T-Shirt Design Contest
/by Christine KellyAs part of our upcoming Awareness Campaign, we are excited to announce a t-shirt design contest! From now until August 12th, you can submit your designs to be considered. Our Board of Directors will vote on the winning design on August 13.
Chelsea’s Hope is celebrating 15 years as a patient advocacy organization this year. We aim to improve the lives of those affected by Lafora disease and help accelerate the development of treatments. Our 2024 Chelsea’s Hope Awareness Campaign aims to spread awareness about Lafora disease and fundraise for the ION283 Safety Study.
What will the shirts be for?
They are for our community to feel connected and raise awareness about Lafora disease. Profits from the t-shirt sales will fund our mission and they will be a prize for fundraisers during our upcoming awareness campaign.
The contest winner will receive a shirt free of charge.
Eligibility
There is no entry fee to submit a design; the contest is open to all. There are no limits on how many designs you submit. However, if you are under 18, a parent or guardian must submit it on your behalf.
Rules and Guidelines
Required Design Components
The organization will place the winning design on the front of the 2024 Awareness Campaign t-shirt, which will be white. The design theme should include “Fight Lafora” and the Chelsea’s Hope logo.
Below are some brand guidelines:
LOGO:
Click here to access the Chelsea’s Hope logo.
DESIGN SOFTWARE:
Your choice, so long you can generate a PDF or image to submit.
COLORS:
The design can include the following colors:
FONTS:
We use Arial and Calibri along with these fonts from Canva:
How We Will Select a Winner
The Chelsea’s Hope Board of Directors will vote on designs, which the Executive Director will present anonymously.
Designs will be reviewed for:
Fine Print of the T-Shirt Design Contest
Publicity and Agreement: Contest entry constitutes an agreement by the submitting designers to grant Chelsea’s Hope Lafora Children Research Fund a free license of all copyrights of the submitted works in perpetuity for publicity and promotional purposes. The winner(s) will be subject to approval by the organization.
Media Release: By submitting artwork, you agree that if your design wins, Chelsea’s Hope Lafora Children Research Fund retains first printing rights and a free license, in perpetuity, to utilize the design on t-shirts and other promotional items and marketing and fundraising materials. You guarantee that your design does not contain any copyrighted material, including images/copy found on the internet, unless marked as published under a Creative Commons (cc) license. Contest entrants not selected as the winning designer(s) retain all rights to their artwork.
G-Tube Tips – A Mother’s Perspective
/by Niki MarkouA Mother’s Perspective on the Benefits of a Feeding Tube for Lafora Disease Patients
Has your child got a Gastrostomy tube, or have you been advised they will need it?
It is a very daunting decision to make as you are still grieving from the devastating diagnosis and watching your child change so rapidly. Take the time to research gastronomy tubes (g-tube) and decide when you feel ready. It is another big change for you, the family, and especially your child. Here are a few recommendations I’d like to share to help make this decision and to make it easier if and where possible.
The benefits of getting a gastrostomy tube inserted early can be helpful as your child is less at risk when their Lafora symptoms are less severe. Having the g-tube early will save on trips to the hospital if there are emergencies where you need to administer emergency medications. You can save the stress by helping your child in the comfort of your own home. You can also give additional nutrition if they are not adequately meeting their dietary requirements. A g-tube will help keep them strong for a lot longer, especially if you can provide them with nutrition that they necessarily wouldn’t eat if you asked them to.
Tips for caring for your child with a gastrostomy tube:
More information on g-tubes
Our Resources page has links to helpful tips. More information on ways to care for your child with a gastrostomy tube can also be found here. You can also join the TubeFed community on Facebook. Finally, check out these resources I’ve found helpful.
My recommendations are from my own experience with my daughter and shared from a mother’s/carer’s point of view. Please discuss everything with your healthcare team and follow any medical practitioner’s advice when given.
Sending Love
Niki
Parent and Director of Family Support