News

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

News / January 22, 2021

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 »

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

News / 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 …

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

In Memorial or Honorarium – October 2020

In Memory or In Honor Donation, News / 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 …

In Memorial or Honorarium – October 2020 Read More »

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

News / 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 …

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

Patient Focused Drug Development Meeting on Krabbe Disease

News / 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 …

Patient Focused Drug Development Meeting on Krabbe Disease Read More »

Minoryx Nexus Clinical Study for cALD

News / May 13, 2020

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 with early cerebral MRI lesions. …

Minoryx Nexus Clinical Study for cALD Read More »

#GivingTuesdayNow is May 5, 2020

News / May 4, 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 nonprofits around the world. At …

#GivingTuesdayNow is May 5, 2020 Read More »

Life During the Covid-19 Crisis: A Chat with Dr. Bernard

News / April 23, 2020

by The Montreal Children’s Hospital Foundation Dr. Geneviève Bernard is a world-renowned expert on genetically determined white matter disorders, called leukodystrophies. Patients are referred to her clinic from all over the world. She brings much comfort to families everywhere who are suffering from these diseases for which the vast majority currently have no known cure. …

Life During the Covid-19 Crisis: A Chat with Dr. Bernard Read More »