#LaforaBodyDiseaseDay Fight Lafora #FightLafora Help us reach a cure!

Lafora Body Disease Day 2024

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This 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:

  • Share your story in your local news or social media and use #FightLafora, #LaforaBodyDiseaseDay, #LaforaAwareness24. Here is a toolkit to help you. You can also invite others to join our movement!
  • Donate or start a fundraiser for the ION283 Safety Study. Thank you to everyone who is already participating.

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: 

#LaforaBodyDiseaseDay Fight Lafora #FightLafora Help us reach a cure!
#LaforaBodyDiseaseDay Fight Lafora #FightLafora Help us reach a cure!

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. Linda and Chelsea

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

I want to join!

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.

Text says 'ION283 Safety Study ASO Therapy for Lafora Disease' over a background image of the Dallas, Texas, skyline

ION283 Safety Study on ClincialTrials.Gov

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A 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:

1) Patients must be between 10-18 years of age at the time of enrollment (meaning they are at least 10 years old and have not yet turned 19 years old)
2) Patients must have a genetically confirmed diagnosis showing a mutation in either EPM2A or EPM2B/NHLRC1
3) Patients must be able to walk independently (meaning they can walk 10 steps without assistance)

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.

Read the protocol
What if my country does not have a patient organization?
If you do not have a patient organization in your country, please contact Chelsea’s Hope at info@chealseashope.org. We will make sure you receive the application packet as soon as it is available.
How will I know when the enrollment process starts?
The enrollment process will open as soon as the patient organizations send out the application packet. The release date and time for the application packet will be announced in advance so that eligible families and their neurologists know when to expect the application.

How will the enrollment process work?
Neurologists must nominate their patient for the study. In order to do that, they will have to complete the application packet distributed by the patient organizations. The release date for this application packet will be announced in advance, and the packet will be sent to all current patient families and neurologists on our contact registry, and to any new neurologists or family members who contact the organization expressing interest in the study.

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

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Noventia Pharma released a statement for the Lafora community that Dr. Gentry read at the 2024 Lafora Disease Science Symposium. Please read it below:

Dear 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

 

The background is bright yellow. On the left is a child with blue face paint. To the right is another child, who has red, white, and black face paint on, Between the children, the text reads, 'FACE it To make childhood dementia impossible to ignore'.

Chelsea’s Hope Joins FACE It 2024

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Wednesday, 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.

The background is bright yellow. On the left is a child with blue face paint. To the right is another child, who has red, white, and black face paint on, Between the children, the text reads, 'FACE it To make childhood dementia impossible to ignore'.


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?

I’m in!