Chelsea’s Hope Announces Launch of Advisory Board

/by

As announced during our 15-year celebratory dinner, Chelsea’s Hope is pleased to share the launch of our Advisory Board.

Chelsea’s Hope has a dedicated Board of Directors, staff, and volunteers who work to execute our mission. To accelerate our path to treatment and expand our capacity to serve families with Lafora, the Board nominated 11 champions in the fields of basic science research, clinical care, and regulatory expertise to provide advice and guidance as we work toward the common goal of a cure for Lafora. We are pleased to share that all 11 nominees have agreed to serve as members of the Advisory Board.

Chelsea's Hope Advisory Board standing in front of a screen

The first Chelsea’s Hope Advisory Board meeting was on September 25th, 2024.

Please join us in celebrating our inaugural Advisory Board: Dr. Pascual Sanz (Valencia Institute of Biomedicine, CSIC), Dr. Souad Messahel (Elpida Therapeutics), Dr. Jose Serratosa (Fundacion Jimenez Diaz University Hospital), Dr. Jordi Duran (IQS Barcelona – Universitat Ramon Llull), Dr. Roberto Michelucci (IRCCS Istituto delle Scienze Neurologiche di Bologna), Dr. Francesca Bisulli (University of Bologna), Dr. Matthew Gentry (University of Florida), Dr. Antonio Delgado-Escueta (David Geffen School of Medicine at UCLA), Dr. Viet Nguyen (Chapman University), Dr. Berge Minassian (UT Southwestern Medical Center), and Mr. Jeff Milton (La Jolla Labs).

The Advisory Board will meet twice yearly and offer guidance and support to the Chelsea’s Hope Board of Directors. We appreciate their advice on our programs, fundraising, and events. 

The inaugural Advisory Board meeting was held on September 25th after our 2024 Symposium. We look forward to these experts’ impact as they work more closely with our organization and advance our mission.

Q&A Session about Family Support for the Safety Study with Dr. Souad Messahel

Announcing a Q&A Session about Family Support for the Safety Study

/by

We are excited to announce a Q&A Session on Zoom about family support for the Safety Study this coming Monday, October 14th, at 10 am ET. Dr. Souad Messahel will join us to answer families’ questions about visas, travel insurance, health insurance, and accommodations for patients accepted into the safety study.

During the meeting, Dr. Messahel will present all the support materials she has for families and answer our questions. Add the event to your Google Calendar, Outlook Calendar, or Yahoo Calendar. Please find the Zoom invite below.

Please email your questions beforehand to Dr. Kit Donohue with the subject line “Safety Study Q&A Question.”

Can’t make it? No worries! We will record the event and share it with families. Also, after the Q&A, Chelsea’s Hope will update our Safety Study FAQs page with everything we’ve learned.

Chelsea’s Hope is inviting you to a scheduled Zoom meeting.

Topic: Q&A Session about Family Support for the Safety Study

Time: Oct 14, 2024 10:00 AM Eastern Time (US and Canada)

Join Zoom Meeting: https://us06web.zoom.us/j/81449041564?pwd=N4umHP5bsNVUO67WrwasK8EbSpncI9.1

Meeting ID: 814 4904 1564

Passcode: 847627

—

One tap mobile

+13092053325,,81449041564#,,,,*847627# US

+13126266799,,81449041564#,,,,*847627# US (Chicago)

—

Dial by your location

• +1 309 205 3325 US

• +1 312 626 6799 US (Chicago)

• +1 646 931 3860 US

• +1 929 205 6099 US (New York)

• +1 301 715 8592 US (Washington DC)

• +1 305 224 1968 US

• +1 253 205 0468 US

• +1 253 215 8782 US (Tacoma)

• +1 346 248 7799 US (Houston)

• +1 360 209 5623 US

• +1 386 347 5053 US

• +1 507 473 4847 US

• +1 564 217 2000 US

• +1 669 444 9171 US

• +1 669 900 6833 US (San Jose)

• +1 689 278 1000 US

• +1 719 359 4580 US

Meeting ID: 814 4904 1564

Passcode: 847627

Find your local number: https://us06web.zoom.us/u/kcY1b48NiF

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

Lafora Body Disease Day 2024

/by

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

/by

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.