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Latest news
In honor of Rare Disease Day, we have come together to raise funds for type-specific programming. This shirt sale is made possible by a collaborative…
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…
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,…
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…
The ULF received donations in memorial or honorarium for the following during the month of October 2020. We apologize for any inadvertent omittance or misspellings.…
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…
Share your story
By Alexandra Worth Axel Riordan was born October 15, 2017. He was a healthy growing boy, hitting all his milestones on time. Around 18 months…
Being part of a family that has endured the devastation of ALD since 1959 when my mother’s brother lost his battle and then losing both…
by Shannon Reed Amera is our sweet 3 year old daughter who battles an extremely rare genetic disease called ACER3 related early childhood onset progressive …