ALD Connect

GREAT NEWS: The European Commission has granted marketing authorization for NEZGLYAL® (leriglitazone), making it the first pharmacological treatment approved for cerebral adrenoleukodystrophy (cALD) in the European Union.

NEZGLYAL is approved for boys with ALD ages 2 to 12 who have cerebral lesions that are not enhancing with gadolinium (Gd-negative) on brain MRI and have a Neurologic Function Score (NFS) of 0 or 1.

This is an important milestone for the ALD community and represents a new treatment option for some boys with cerebral ALD.

We are grateful to the researchers, clinicians, industry partners, patients, and families who have contributed to advancing ALD research and making progress like this possible.

Congratulations to Minoryx Therapeutics and Neuraxpharm, and to the entire ALD community! 💙

Press release: https://www.minoryx.com/media/european-commission-grants-marketing-authorisation-for-nezglyal%C2%AE-(leriglitazone)-the-first-pharmacological-treatment-approved-for-cerebral-adrenoleukodystrophy-(cald)-a-rare-neurodegenerative-disease/

Life with ALD can bring plenty of unexpected twists and turns. Join us for a positive, supportive conversation about navigating the things we did not see coming, managing difficult emotions, building resilience, and finding ways to move forward when plans change. Connect with others who understand and pick up practical strategies for handling whatever lands on your BINGO card next!

Register here: https://lp.constantcontactpages.com/ev/reg/qbufevp?mode=preview&source_id=59c32fb3-290d-4d36-9f49-26a8b9816c05&source_type=em&c=

This September, you can help raise awareness of ALD. 💙

ALD Connect is joining leukodystrophy organizations around the world in going blue for Leukodystrophy Awareness Month.

Our ALD Makes Me Blue campaign is live! Wear blue, share your photos, and tell others why ALD awareness matters.

Want to do even more? Your donation helps ALD Connect continue our work for individuals and families affected by ALD.

Donate here: https://givebutter.com/aldmakesmeblue2026

Together, we can make a difference for the ALD community.

ALD Makes Me Blue By ALD Connect

Our Patient-Led Planning Committee brings together members of the ALD community to share their experiences, identify priorities, and help shape programs, resources, and events around what matters most to patients and families.

By involving the people most affected by ALD throughout the planning process, we can make better decisions and create more meaningful opportunities for connection, education, and support.

Join ALD Connect board members and fellow community members at our next virtual meeting on September 16. We'll be discussing our auction and other ongoing initiatives. The committee will also meet in person at our 2026 Annual Meeting and Patient Learning Academy.

Register for next week's meeting here: https://aldconnect.org/event/patient-led-planning-committee-4/

We are proud to share our new Pathway to a Cure page! Pathway to a Cure is ALD Connect’s patient-prioritized research agenda for accelerating progress toward better treatments and, ultimately, a cure for ALD.

A patient-prioritized research agenda helps ensure that research is guided by the needs, experiences, and questions of the people most affected by ALD. It ensures research reflects what matters most to patients and families, helps direct limited resources toward the greatest unmet needs, and keeps the community focused on outcomes that can meaningfully improve people’s lives.

Important work is happening across the ALD community, but it is not always easy to see how the different pieces connect. All stakeholders deserve greater visibility into the research underway, the progress being made, and the questions that still need to be answered.

Pathway to a Cure helps us see the bigger picture. A shared direction and clear destination will help us navigate each turn, build bridges, open new routes, connect promising ideas, and continue moving toward a cure, even when the journey includes unexpected detours or important stops along the way. By mapping the pathway, we can identify priorities and determine where greater attention, collaboration, and investment are needed.

Over the coming months, we will highlight work from members of our Collaborative Research Network to show how their research contributes to the pathway. The pathway will continue to evolve, and we will update the page regularly, so be sure to check back often.

There will also be opportunities for patients, families, researchers, clinicians, donors, industry partners, and advocates to contribute through surveys, focus groups, community discussions, and other collaborative initiatives. We invite you to join us in accelerating meaningful progress toward a cure! 💙

https://aldconnect.org/clinical-trials-and-research/pathway-to-a-cure-for-adrenoleukodystrophy-ald/

We love seeing ALD Connect(ions)! Being with people who truly “get it” can make such a difference.

During Leukodystrophy Awareness Month, we encourage you to meet someone new in the community, reconnect with an old friend, or spend time with others who understand the ALD journey. If you are planning to get together with someone else in the community, let us know. We may be able to assist with eligible expenses.

Looking for an easy way to get connected? Join us at our 2026 Annual Meeting and Patient Learning Academy in November, where you can meet other families, share experiences, and build meaningful relationships within the ALD community.

Make a connection, and be sure to share it with us! 💙

Whether ALD has been part of your life for generations, entered it unexpectedly, or is something you are learning about for the first time, your voice matters. During Leukodystrophy Awareness Month, we invite everyone affected by ALD, along with families, friends, clinicians, researchers, and supporters, to stand together as one community. Share your story below and help others understand the many ways ALD touches lives. 💙

Adrenoleukodystrophy (ALD) is a rare genetic disorder that many people have never heard of until it touches their own life or someone they know. ALD is caused by mutations in the ABCD1 gene, which normally helps break down very long-chain fatty acids (VLCFAs). When this process does not work properly, VLCFAs build up and damage the brain, spinal cord, and adrenal glands. An ALD diagnosis brings uncertainty, since there is currently no way to predict how the disease will manifest or progress in each patient.

During Leukodystrophy Awareness Month, we encourage you to share how ALD has touched your life. We also invite others to stand with our community. A heart on this post is an easy way to let individuals living with ALD know you see them and care. 💙

Join us tonight! Come with a list of things you want others to know about what it is like to live with ALD. We want to hear what matters most to you and what you wish others better understood.

https://aldconnect.org/get-involved/community-calendar/

Our upcoming Structured Mental Health Community Call, originally scheduled for Thursday, August 20th, has been rescheduled to Thursday, August 27.

What do you wish people without ALD understood?

Living with ALD can feel isolating, particularly when so much of what you are carrying is invisible to others. The uncertainty. The anxiety around monitoring and MRIs. The impact on relationships and family. The emotional toll of living with a disease that others may not fully understand.

There may be other things you wish people knew, too. The perspective you’ve gained. The strength you’ve discovered. The relationships that have deepened. The moments of joy, gratitude, humor, or connection that are also part of life with ALD.

Join us on August 27th for an open conversation about the realities of living with ALD and connect with others who understand.

See you next week!

https://lp.constantcontactpages.com/ev/reg/sug4946

Join us tonight! Come with a list of things you want others to know about what it is really like to live with ALD. We want to hear what matters most to you and what you wish others better understood.

https://aldconnect.org/get-involved/community-calendar/

Our upcoming Structured Mental Health Community Call, originally scheduled for Thursday, August 20th, has been rescheduled to Thursday, August 27.

What do you wish people without ALD understood?

Living with ALD can feel isolating, particularly when so much of what you are carrying is invisible to others. The uncertainty. The anxiety around monitoring and MRIs. The impact on relationships and family. The emotional toll of living with a disease that others may not fully understand.

There may be other things you wish people knew, too. The perspective you’ve gained. The strength you’ve discovered. The relationships that have deepened. The moments of joy, gratitude, humor, or connection that are also part of life with ALD.

Join us on August 27th for an open conversation about the realities of living with ALD and connect with others who understand.

See you next week!

https://lp.constantcontactpages.com/ev/reg/sug4946