ALD Connect

We're excited to announce that Taylor Kane will be serving as a consultant supporting the Global ALD Advocacy Network.

Taylor is a patient engagement and advocacy consultant with more than 15 years of experience championing the needs of the adrenoleukodystrophy (ALD) and rare disease communities and serving as a trusted intermediary between patients, researchers, and industry. Her work is shaped by both professional expertise and lived experience as a presymptomatic woman with ALD. Now based in Europe, Taylor also brings an important international perspective that will help strengthen our global collaborations.

Following the loss of her father to ALD, Taylor became deeply involved in rare disease advocacy, including helping advance legislation establishing newborn screening for ALD in New Jersey. She is currently working with New Jersey legislators on legislation that would require health insurance companies to cover IVF with preimplantation genetic testing (PGT-M) for people at risk of passing on severe genetic conditions.

Taylor has collaborated with a wide range of for-profit and nonprofit organizations, serving as a consultant to companies across the United States, Europe, and Latin America. She guides drug developers, genomics companies, reproductive health organizations, and IVF clinics in building meaningful partnerships with the patient communities they serve.

Taylor is also the founder and consulting executive director of Remember The Girls, an international nonprofit organization dedicated to supporting females with X-linked genetic conditions. ALD Connect and Remember The Girls have partnered on several initiatives over the years.

Taylor will help strengthen our relationships with advocacy organizations around the world, expand international collaboration, and ensure the perspectives of patients and families remain central to our global initiatives. Her experience, international perspective, and strong relationships across the rare disease community will be instrumental as we continue building a stronger, more connected global ALD community.

Our 2026 Annual Meeting & Patient Learning Academy will be held November 6-7 in Salt Lake City, Utah!

We're looking forward to bringing together patients, families, caregivers, researchers, physicians, industry partners, advocates, and donors from around the world for two days of learning, collaboration, and connection.

The festivities begin Thursday evening with a casual gathering for patients and families hosted by our ALD Connect Ambassadors. On Friday and Saturday, attendees will hear the latest updates on on our Breakthrough Research Fund, Collaborative Research Network, and exciting new initiatives. We'll have presentations from international experts and Emerging Investigators, a patient panel, an Ask the Experts session, poster presentations, and plenty of opportunities to engage with others in the global ALD community.

Register now: https://aldconnect.org/get-involved/annual-meeting-and-patient-learning-academy/

BREAKING NEWS: Today marks an important milestone for the ALD community.

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorization, under exceptional circumstances, for leriglitazone (NEZGLYAL®) for the treatment of cerebral adrenoleukodystrophy (cALD) in boys aged 2-12 years with non-gadolinium enhancing (Gd-negative) lesions.

The CHMP opinion is an important step in the European regulatory process. The final decision on marketing authorization rests with the European Commission, which is expected to issue its decision in the coming months.

We are grateful to every patient, family, researcher, clinician, study coordinator, and advocate who has contributed to ALD research. Advances like this are only possible because of the dedication of an entire community committed to improving outcomes for people living with ALD.

Read the full press release below. 💙

*Proposed indication included in the CHMP positive opinion: NEZGLYAL® is indicated for the treatment of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2-12 years with non-Gadolinium enhancing (i.e. Gd-negative) lesions in brain MRI and Neurological Functional Score (NFS) of 0 or 1. Please note: The proposed indication is based on the CHMP positive opinion and remains subject to final approval by the European Commission.

NEZGLYAL® (leriglitazone) receives a positive CHMP opinion for the treatment of cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease News Home Media NEZGLYAL® (leriglitazone) receives a positive CHMP opinion for the treatment of cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease

What does it take to cure ALD?

A cure will require progress across the entire research continuum, from understanding the biology of disease to developing new therapies, improving clinical care, slowing or preventing disease progression, preventing disease before symptoms occur, and ultimately changing the future for individuals and families affected by ALD.

That's why ALD Connect is developing a living roadmap of the global ALD research landscape: Pathway to a Cure.

Leveraging the expertise of our Collaborative Research Network and partners across the ALD community, we are creating the first comprehensive view of ALD research by mapping scientific activity across every stage of discovery and development. This roadmap will highlight where progress is being made, identify gaps and unmet needs, uncover opportunities for collaboration, and help reduce unnecessary duplication of effort. It is designed to support researchers, clinicians, industry partners, advocacy organizations, donors, and families by providing a clearer picture of where the field is today and what it will take to move it forward.

The Pathway to a Cure will also help inform strategic fundraising and investment by identifying where resources can have the greatest impact. Every discovery builds on the one before it, and by connecting the entire research ecosystem, we can help accelerate the development of better treatments and, ultimately, a cure.

Because the fastest path to a cure is one we build together. 💙

Join us for our Structured Mental Health call tomorrow: Everything is good, so why do I feel bad?

Even when life is going well, many people affected by ALD still experience anxiety, sadness, or a sense that something bad is just around the corner. Join us as we discuss why these feelings can persist during times of stability, explore the emotional impact of living with both hope AND uncertainty — all while sharing practical strategies for coping with worry while allowing ourselves to embrace the good moments.

Register here: https://lp.constantcontactpages.com/ev/reg/jv9jazc

ALD Connect is investing in the future of ALD research through grant opportunities that accelerate discovery, strengthen collaboration, and support innovative research.

Our current funding opportunities include Collaborative Research Network (CRN) grants, which support multidisciplinary teams working together to address the highest priorities in ALD, and Emerging Investigator grants, designed to help launch promising research careers.

Thank you to everyone who has supported our Breakthrough Research Fund. Your generosity makes these opportunities possible.

We look forward to reviewing this year's applications and supporting research that brings us closer to better treatments and improved outcomes for everyone affected by ALD.

Donate: https://aldconnect.org/donate/

At ALD Connect, we believe that global collaboration is essential to advancing research, improving access to high-quality care, accelerating the development of new therapies, and finding effective treatments for every phenotype of ALD. By bringing together advocates from across the globe, we can strengthen our collective efforts and advance progress for individuals and families affected by ALD.

We are thrilled to partner with Amy Gaviglio, MS, CGC, Founder and CEO of Connetics Consulting, LLC. She will help establish and guide the Global ALD Advocacy Network. Amy is an internationally recognized genetic counselor and leader in newborn screening, rare disease policy, implementation, and advocacy.

Through Connetics Consulting, Amy advises organizations around the world on newborn screening and rare disease strategy. She currently collaborates with the Centers for Disease Control and Prevention (CDC), the Association of Public Health Laboratories (APHL), Expecting Health, Primary Health, and numerous other organizations. She serves as Co-Chair of APHL's New Disorders in Newborn Screening Subcommittee, Vice Chair of the Newborn Screening Expert Panel for the Clinical and Laboratory Standards Institute (CLSI), a member of the Scientific Advisory Board for the MPS Society, a member of the Executive Board of the International Society for Neonatal Screening, and Chair of the Board of Directors for the EveryLife Foundation for Rare Diseases.

We look forward to empowering advocates, strengthening partnerships, and helping organizations make an even greater impact for individuals and families affected by ALD. 💙

We had a great time at the United Leukodystrophy Foundation's conference! Thank you to the ULF for bringing the leukodystrophy community together. We were reminded of the power of community and excited to see some of our work highlighted.

We hope you will join us for our 2026 Annual Meeting and Patient Learning Academy on November 6-7th in Salt Lake City, Utah! Don't wait to register - our room block is filling up!

https://aldconnect.org/get-involved/annual-meeting-and-patient-learning-academy/

We're at the United Leukodystrophy Foundation conference this week! Yesterday's scientific sessions were outstanding, bringing together researchers and clinicians to share the latest advances in leukodystrophy research. Several ALD Connect Board Members are presenting as part of the ALD session this afternoon. We're looking forward to connecting, learning, and continuing important conversations that move the field forward. 💙

ALD Connect Board Member, Jesse Torrey, has a way with words.

In her latest blog post, she reflects on the incredible ALD Connect(ions) gathering her family hosted this past weekend and the moments that made it so meaningful. Her reflections capture what so many families experience when they come together: a sense of belonging, understanding, and hope.

Thank you to Jesse and her family for opening their home and helping strengthen our community. Thank you to everyone who spent the day building connections and supporting one another.

We hope you'll take a few minutes to read her beautiful recap of a memorable day. If you're interested in hosting your own ALD Connect(ions) event, please email [email protected].

https://smilesandducttape.com/