LBSL Voice

LBSL is ultra-rare, complex, and not fully documented in the medical literature.  The key to understanding and eventually curing this disorder is to listen carefully to the people living with it every day.  We strongly believe that it is critical for clinicians, researchers, and regulators to know about the lived experience and priorities of this community.  ​

It is a primary mission of Cure LBSL to:​

  • Amplify the patient voice in the medical and scientific communities​

  • Facilitate patient engagement and advocacy efforts with the FDA​

Please join us by sharing your voice, sharing your priorities, and sharing your stories.  You will be heard!

Research Opportunity

Cure LBSL Quality of Life Research Project

The Cure LBSL Quality of Life Research Program aims to understand the unique challenges faced by individuals with Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and Lactate Elevation (LBSL) and their caregivers.

This study seeks to gather comprehensive quality of life data to support future research and improve support for those affected by LBSL.

What is included?

  • Polls at the live Cure LBSL Conference July 26-29.

  • Zoom interviews with LBSL research staff starting in August 2024.

Why is this study important?

  • Knowledge gained may help researchers and doctors understand more about the quality of life of people with LBSL

Who can participate?

  • Anyone with LBSL or who cares for someone with LBSL.

Where can I join?

Principal Investigator: Dr. Danielle Boyce
Research approved by North Star Review Board

FDA Listening Session

Cure LBSL has been advocating for the patient voice to be heard with the US Food and Drug Administration (FDA). We are so pleased that they have accepted our request for a Patient Listening Session, which was held on Tuesday, May 30, 2023. For more information, please visit our Advocacy page.

On February 18, 2023, we held an LBSL Community Meeting to share information about the upcoming FDA Patient Listening Session. ​

For more information about FDA Patient Listening Sessions, please visit:​ https://www.fda.gov/patients/learn-about-fda-patient-engagement/fda-patient-listening-sessions

Share Your Voice

Based on the specific requirements from the FDA for the Listening Session, only a small number of individuals will be able to participate live at the meeting.  However, everyone who has shared during our patient voice sessions will have their voice heard and their stories told in some form during the presentations.  We also hope to engage with the FDA at other types of meetings in the future, where additional participants will be included.  Thank you for your understanding!​

The entire LBSL community is invited to share their stories and voice for this event.  All patients, parents, siblings, and caregivers may: ​

Patient Voice Meetings

We are scheduling several different opportunities for you to share your stories and your voices in preparation for our Patient Listening Session with the FDA.  There will be different conversation prompts and discussion topics at each session, and you are welcome to join as many sessions as you would like.  We may add additional sessions as needed.  ​

  • Please register in advance so that we know who to expect (you may always cancel later, but it helps us for planning).  ​

  • These meetings will be held in English. Please also let us know if you have any language or other accessibility needs. If there is enough interest, we will schedule meetings in languages other than English.  ​

  • IMPORTANT: all sessions will be recorded and potentially shared during a semi-private LBSL Patient Listening Session with the FDA. If you do not consent to being recorded, please do not join these calls.​

  • Additional voluntary consent may be requested to share recordings on our website and/or on social media.​

  • To protect the privacy of our community, these patient voice sessions are by invitation only. 

Scheduled Patient Voice Zoom Meetings​

  • Saturday, Feb 18, 2023 03:00 PM Eastern Time – English​

  • Thursday, Feb 23, 2023 08:00 PM Eastern Time – English​

  • Friday,  Feb 24, 2023 08:00 AM Eastern Time – English​

  • Sunday, Feb 26, 2023  6:00 PM Brasilia Time – PORTUGUESE​

  • Sunday, Feb 26, 2023 06:00 PM Eastern Time – English​

  • Friday: Mar 3, 2023 12:00 PM Eastern Time – English​

  • Additional times and dates may be added as needed​

Please join the LBSL International Contact List to be invited to patient voice sessions. CLICK HERE