News & Blog

IONIS Statement for the Lafora Community September 4, 2024

Ionis Statement for the Lafora Community

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IONIS logoIonis shared a statement with Chelsea’s Hope for the Lafora community. They have agreed to license the ION283 program to Noventia Pharma, and Noventia will assume responsibility for developing it. 

The 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.

Read statement

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 Approval ASO Therapy for Lafora Disease

ION283 Safety Study Approved

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We 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.

Berge A. Minassian, M.D

“30 years in the making, we now have FDA approval to start our ION283 clinical trial.

I would like to thank each of your associations for your support over the years…” – Dr. Berge Minassian

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.

Donate today!


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.”

Purple text says: T-SHIRT DESIGN CONTEST 2024. The background is purple. The Chelsea's Hope round logo is to the bottom right. There's a photo of a white t-shirt in the center.

Announcing a 2024 T-Shirt Design Contest

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As 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.

Submit designs

White text says: T-SHIRT DESIGN CONTEST 2024. The background is purple. The Chelsea's Hope round logo is to the right of the center, separating the text (left) from an image of a white t-shirt (right).T-Shirt Design Contest Guidelines

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

  1. You must create an original design and incorporate all of the required design components outlined below. The design must align with the Chelsea’s Hope Lafora Children Research Fund brand. Your submission may not have been previously published and must not contain profanity, trademarks, other organization’s logos, or any copyrighted works of any other person or business.
  2. Design(s) must be submitted via the submission form by 11:59 pm (PT) on August 12, 2024.
  3. The winning designer agrees to sign a waiver allowing Chelsea’s Hope to use their design free of charge for any and all printing and promotional purposes.
  4. The contest winner must agree to submit a high-resolution art file within five days of notification and agree to work with the printer if slight changes/modifications are necessary for production. If not, the organization might choose an alternate winner to meet our production deadlines.
  5. By submitting an entry, you are agreeing to all contest rules. If you have any questions, please email info@chelseashope.org.

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: 

Six circles of different colors, labeled with their hex color codes. From left to right, they are: #aa499d, #505050, #78276d, #218a7c, #7547d1, #cf87c5.

 

 

FONTS:

We use Arial and Calibri along with these fonts from Canva:

There are two rows of text names of fonts. The text is in its named font. Row one, left to right: Centrale Sans Medium, Neue Machina, Apricots. Row two, left to right: Centrale Sans Light Light, Mansalva, Brittany, Antonio.

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: 

  • Concept and originality of design
  • Visibility from a reasonable distance
  • Adherence to design and submission rules and guidelines
  • Adherence to the Chelsea’s Hope branding guidelines, including logo usage, colors, and typefaces
  • Highlighting the organization’s mission

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.

 

Blog post G-Tube Tips for Lafora families

G-Tube Tips – A Mother’s Perspective

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G-Tube Tips for Lafora Disease Patients blog

A 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:

  1. A low-fiber formula for nutrition is ideal, as a formula with high fiber is hard for a slow gut to process. A high-fiber formula could cause more frequent constipation and vomiting issues as their bodies cannot break down the fiber quickly enough. A high protein feed with a good water intake is helpful. 
  2. Stool softeners can alleviate any adverse effects, like constipation and vomiting.
  3. If lying down, elevate their head to a 30–45-degree elevation to avoid aspiration into their lungs.
  4. If you can administer the formula slowly or intermittently, then this will help them absorb it and avoid overfill, which can cause vomiting. 
  5. Overnight feeds can help if they get upset with having to be fed this way and put less stress on the body to work quickly. 
  6. Venting/Degassing is good if they are having painful gas issues.

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