2020 - United Leukodystrophy Foundation

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

News / December 21, 2020 December 21, 2020

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), a lentiviral vector-based gene therapy …

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In Memorial or Honorarium – November 2020

In Memory or In Honor Donation / December 21, 2020 December 21, 2020

The ULF received donations in memorial or honorarium for the following during the month of November 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 Alex Bouley Bernard Levine, M.D. Aryabella HaegerShannon Wright Daxton WallaceSandy Wallace Emily CullenLisa Cullen …

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In Memorial or Honorarium – October 2020

In Memory or In Honor Donation, News / November 1, 2020 February 6, 2021

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. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF C. Michael Jaynes: Angela Pontius, C. Michael Jaynes – All those fighting leukodystrophies and …

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In Memorial or Honorarium – September 2020

In Memory or In Honor Donation / October 4, 2020 October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of September 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 Ella McKee: Heather McKee – For our brave daughter who continues to fit her …

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In Memorial or Honorarium – August 2020

In Memory or In Honor Donation, uncategorized / September 6, 2020 October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of August 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 Amie Matlon: Marianne Matlon Aveline Brendel: Pauline Naudin, William Brendel, Amy McKenzie, Ryan McKenzie, …

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In Memorial or Honorarium – July 2020

In Memory or In Honor Donation, uncategorized / August 9, 2020 October 4, 2020

The ULF received donations in memorial or honorarium for the following during the month of July 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 David Stocki: Melissa Scier – For my son who has Alexander Disease Leukodystrophy Dax …

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In Memorial or Honorarium – June 2020

In Memory or In Honor Donation / July 14, 2020 July 14, 2020

The ULF received donations in memorial or honorarium for the following during the month of June 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 Angelica Gallegos: Rozzana Vasquez Cobos Anna Walentowski: Anonymous Briana Singh: Stephanie Singh Dax Wallace: …

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Newborn Screening Saves Lives Reauthorization

News / June 23, 2020 June 23, 2020

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 and the storage of dried …

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Research Study: Impact of COVID-19 on People Living with Rare Diseases and Their Families

News / June 22, 2020 June 22, 2020

The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. We want to learn from you and hope that you are willing to share your experience. The Rare Diseases Clinical Research Network’s (RDCRN) Data Management and Coordinating Center, which is supported …

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Patient Focused Drug Development Meeting on Krabbe Disease

News / June 8, 2020 June 8, 2020

NORD invites you to register for an externally-led Patient-Focused Drug Development (EL- PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30 – 4:30 pm ET. Patients and caregivers impacted by Krabbe disease who are interested in potentially speaking at the meeting as panelists should be sure to fill out this form by August 7. If you …

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