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We are Hiring!

Are you experienced at development and fundraising and looking to work for a mission driven organization? With the ability to work remotely, the United Leukodystrophy Foundation (ULF) is seeking a dynamic Executive Director to embrace and move the Strategic Plan forward, side by side with dedicated and experienced staff members.

Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD)

Interactions with FDA anticipated by mid-2021 to determine the BLA filing strategy for OTL-200 BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics, a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200,…

Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

Press Release: Orchard Therapeutics BOSTON/LONDON/MILAN, December 21, 2020 – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene),…

Newborn Screening Saves Lives Reauthorization

Thank you ALL for supporting last year’s sign on letter to add language to Newborn Screening Saves Lives Reauthorization which would fund states in order for every state to become RUSP compliant. As we know Newborn Screening Saves Lives Reauthorization is still undergoing challenges with pushback from Senator Rand Paul…

Minoryx Nexus Clinical Study for cALD

Minoryx Therapeutics is recruiting for a clinical trial focusing on cALD in Europe. The NEXUS clinical trial is a phase 2, open-label clinical study designed to assess the efficacy and safety of leriglitazone in male pediatric patients with early stage cerebral X-linked adrenoleukodystrophy (cALD). The NEXUS study recruits cALD patients…

#GivingTuesdayNow is May 5, 2020

Join the Global Day of Giving and Unity! #GivingTuesdayNow is set to take place on May 5, 2020 as an emergency response to the unprecedented need caused by COVID-19. The day is designed to drive an influx of generosity, citizen engagement, business and philanthropy activation, and support for communities and…

A Message about Safety and COVID-19

March 2020  Dear ULF Community,  On behalf of the Board of Directors, Medical and Scientific Advisory Board, and the staff of the ULF, please know that we are following the developments of COVID-19 and feel the concerns of our community. At this time, all we can offer is advice on…

A Letter from the ULF President

Dear Friends and Supporters of the United Leukodystrophy Foundation,  As the President of the ULF I am excited to talk to you about the many changes that are taking place as we build the future of the Foundation. In the past year, we’ve added seven new members to our board…

Embolden Study for MLD

Worldwide, MLD occurs in 1 in 40,000 people, primarily children.  Early symptoms of MLD include trouble walking, impaired speech, and muscle weakness. The Embolden Study is conducting research for infants and children born with metachromatic leukodystrophy. Study overview: The Embolden Study is evaluating the safety and effects of a study drug on…

Magical Memories Disney Raffle 2019

Buy tickets for a chance to win a Disney vacation for 4! Support the United Leukodystrophy Foundation with the purchase of a Magical Memories Disney Raffle ticket. The winner of the raffle will receive $1,050 in Disney gift cards redeemable toward admission, food or souvenirs; 4-night stay in one standard…

2018 Benefit Drawing

The time has come once again for the United Leukodystrophy Foundation’s Annual Benefit Drawing fundraiser! Thanks to the involvement of our members, friends, and donors like you, we are able to continue to award vital funding to researchers, who strive to find new treatments for the leukodystrophies and to provide…

Leukodystrophy and genome sequencing: for genetically undiagnosed patients for the 100,000 genomes project

Henry Houlden, Professor of Neurology and Neurogenetics at the UCL Institute of Neurology and The National Hospital for Neurology and Neurosurgery, Queen Square, London is looking to collect blood samples via mail or at their London hospital. Learn more here: http://bit.ly/2w5RtSe If you are interested in participating, please contact: Henry HouldenProfessor of Neurology and…

2017 Research Grant Awardees

Every year, the ULF puts a call out to the leukodystrophy medical professional community asking for research proposals in need of funding. The number of grant applications have consistently grown in the past years, and this year was no exception. Submissions from all over the country as well as internationally…

2017 Benefit Drawing Winners

This year’s Annual ULF Benefit Drawing winners have been chosen! We had a special Halloween visitor to the office this year to pull the winning tickets: 2-year-old Clark dressed up as Carl from the movie UP! The winners are… • $500 Visa Gift Card – Patrick M. of Virginia• Rolling iPad Stand…

Dr. David Edwin

We at the ULF wish to send our sympathy to the family of one of our main contributors to our annual ULF family conference who passed away in June. His obituary is below. Dr. Edwin has been a contributor addressing the psychological needs of our families and how they can cope with…

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