ULF Awards $33,000 Research Grant to Advance Gene Therapy for H-ABC
The United Leukodystrophy Foundation (ULF) is proud to announce the award of a $33,000 research…
Research Summaries October 2025
ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and…
New Research: Endogenous Repair in Vanishing White Matter
A newly published article, “Endogenous Repair in Vanishing White Matter” explores how the brain may…
The Hidden Impact of Vanishing White Matter (VWM) on Families
New Research Sheds Light on the Burden for Parents, Siblings, and Partners A groundbreaking international…
Positive Results Announced for Alexander Disease Clinical Trial
Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis…
Minoxry Therapeutics: Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cALD
Press Release Summary (December 11, 2024): Minoryx Therapeutics, a late-stage biotech company, and Neuraxpharm Group,…
Viking Therapeutics Announces Results from Phase 1b Clinical Trial of VK0214 in Patients with X-ALD
BridgeBio Shares Positive Data from Canavan Disease Study
BridgeBio Shares Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study of Gene…
ULF Awards $33,000 Research Grant to Advance Gene Therapy for H-ABC
The United Leukodystrophy Foundation (ULF) is proud to announce the award of a $33,000 research…
Historic Expansion of Newborn Screening Includes MLD
Historic Expansion of Newborn Screening Includes Metachromatic Leukodystrophy (MLD) We are encouraged to share that…
Eden’s Story: Helping Families and Supporting Research Through Her Legacy
Eden’s Story: Helping Families and Supporting Research Through Her Legacy https://ulf.org/wp-content/uploads/2025/12/Eden.mp4We are the Murphy Family,…
Hope in Action: ULF Board Member’s Son Receives World’s First SLC6A1 Treatment
We are excited to share a powerful new People Magazine feature highlighting our ULF Board…