Year: 2021

Shari S., ALD

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 my older brother in 1972 and my younger brother in 1979. Knowing since 1987 when I was 18 years old that I am a carrier set the stage for more painful …

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Amera Reed, ACER3

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  Leukodystrophy. There are only about 20 known cases of this type worldwide!  Our daughter was diagnosed with this progressive disease at age 2. She was initially presumptively diagnosed with other …

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In Memorial or Honorarium – January 2021

The ULF received donations in memorial or honorarium for the following during the month of January 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815.748.3211. IN HONOR OF Aryabella Haeger Shannon Wright – In behalf of Star Chaser Crafts  Aveline Brendel Lyndsay …

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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, an investigational ex vivo autologous hematopoietic stem …

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

In Memorial or Honorarium – December 2020

The ULF received donations in memorial or honorarium for the following during the month of December 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815.748.3211. IN HONOR OF Aayaansh ModiAnonymous  Ainsley CunninghamAnonymous Cheryl Malden  Alex Hiles Cynthia Powell  Amari BallDiane Mobeck Ken Mobeck  Ben …

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FDA Clearance of Investigational New Drug for Patients with Krabbe Disease

January 4, 2021 by Dan Salvo Forge Biologics has received IND, IBC, and IRB clearance for FBX-101, a gene therapy for patients with Krabbe disease, a rare and fatal genetic disorder FBX-101 is a first-in-human potential treatment utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene to cells in the CNS and …

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