In Memorial or Honor – March 2022
The ULF received donations in memorial or honor for the following during the month of March 2022. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Ainsley Ouszt John Rosso Catherine Rosso AJ…
In Memorial or Honor – February 2022
The ULF received donations in memorial or honor for the following during the month of February 2022. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Aidan Lodge Susan Lodge Alex Bouley Jane…
In Memorial or Honor – January 2022
The ULF received donations in memorial or honor for the following during the month of January 2022. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Ainsley Ouszt John Rosso Catherine Rosso AJ…
In Memorial or Honor – December 2021
The ULF received donations in memorial or honor for the following during the month of December 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Ainsley Cunningham Elizabeth Mickle LA 77 Alberta…
In Memorial or Honor – November 2021
The ULF received donations in memorial or honor for the following during the month of November 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Adele Moss Claudette Alice Collier Adele Moss…
In Memorial or Honor – October 2021
The ULF received donations in memorial or honor for the following during the month of October 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Aidan Chapleau Mary Ellen Ross Alec Jump…
In Memorial or Honor – September 2021
The ULF received donations in memorial or honor for the following during the month of September 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Aidan Chapleau Keith Colley Brianna Sanchez Kimberly…
Heather & Holly, ALSP
By Heidi Edwards I’m Heidi Edwards, President and founder of Sisters Hope Foundation, a non-profit organization supporting families struggling with Hereditary Diffuse leukodystrophy with spheroids or HDLS. HDLS is now known as Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, or ALSP. I am also a caregiver for my twin…
In Memorial or Honorarium – August 2021
The ULF received donations in memorial or honorarium for the following during the month of August 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Daxton Wallace Sandy Wallace Gregory Brown John…
In Memorial or Honorarium – July 2021
The ULF received donations in memorial or honorarium for the following during the month of July 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Daxton WallaceSandy Wallace Henry CrawleyThomas Carter Ian…
Poxel Announces New Strategic Direction with Increasing Focus on Rare Metabolic Diseases Following Recent Achievements
Download as PDF July 12, 2021 Following the recent approval of TWYMEEG® (Imeglimin) in Japan and associated potential future revenues, Poxel to accelerate and expand rare metabolic disease programs leveraging existing platforms and proven capabilities Poxel to advance its deuterated thiazolidinediones (dTZD) and direct adenosine monophosphate-activated protein kinase (AMPK) activator…
In Memorial or Honorarium – June 2021
The ULF received donations in memorial or honorarium for the following during the month of June 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Ashley WolfJohn Wolf Daxton WallaceSandy Wallace FiaLian…
In Memorial or Honorarium – May 2021
The ULF received donations in memorial or honorarium for the following during the month of May 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211. in honor of Aidan LodgeSusan Lodge Alex HilesJackie Bowman Daxton…
In Memorial or Honorarium – April 2021
The ULF received donations in memorial or honorarium for the following during the month of April 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Aidan ChapleauRobert KiddBarry MacDonaldMarg MacDonald All those fighting leukodystrophies…
Axel Riordan, MLD
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 old Axel started developing problems while walking; locking knees, stumbling, eventually falling a lot. It all progressed slowly at first so at his two-year checkup…
In Memorial or Honorarium – March 2021
The ULF received donations in memorial or honorarium for the following during the month of March 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Alec JumpJames Jump Leesa Jump Alex Bouley Charna Levine Brad…
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…
In Memorial or Honorarium – February 2021
The ULF received donations in memorial or honorarium for the following during the month of February 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Henry Crawley David Crawley – I love you Henry. …
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…
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 [email protected] or by calling 815.748.3211. IN HONOR OF Aryabella Haeger Shannon Wright – In behalf of Star…
We’re All in This Together #RareDiseaseDay
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 effort among these amazing organizations that will help offset future costs of type-specific programming. We are all in this together. Orders will be shipped directly…
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,…
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 [email protected] or by calling 815.748.3211. IN HONOR OF Aayaansh ModiAnonymous Ainsley CunninghamAnonymous Cheryl Malden Alex Hiles Cynthia Powell …
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…
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),…