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 the address that you specify. …
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 …
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 …
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 office@ulf.org or by calling 815.748.3211. IN HONOR OF C. Michael Jaynes: Angela Pontius, C. Michael Jaynes – All those fighting leukodystrophies and …
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 …
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 …
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 »
from BlueCross BlueShield of Illinois Look around and you’ll see caregivers. In the U.S., 1 in 3 adults give care to others. Caregivers may be taking care of elderly parents, a sick spouse or a disabled child. Being a caregiver can be very stressful and demanding. It can even lead to caregiver burnout, a feeling …
Taking Care of Yourself When Taking Care of Others Read More »
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. …
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 …
