Calyx: New Clinical Trial in cALD
Learn more at www.cald-study.com!
News
Vigil Neuro: ALSP Community Letter
The ULF is happy to share a community letter from Vigil regarding their potential treatment option for those affected by ALSP! Here is a preview but click the links below to read the whole letter and press release. “Dear ALSP Community Members: This week we announced positive interim data from IGNITE, our Phase 2 clinical …
ALSP Community Survey: Genetic Testing
Vigil Neuroscience, a biotechnology company researching potential treatments for ALSP, is engaging with the ALSP patient community, caregivers, advocacy groups, and healthcare providers to better understand the needs of community members. Open Health is an independent company engaged by Vigil Neuroscience to conduct a survey to understand behaviors and attitudes towards genetic testing for ALSP in the community. Results from …
TWELVE NEW ICD-10 CODES FOR LEUKODYSTROPHIES
FOR IMMEDIATE RELEASE Today marks a huge leap forward for the leukodystrophy community with the adoption of twelve new diagnostic definitions through the International Classification of Diseases – Tenth Revision. The ICD-10 code system is used by the health field to classify diagnoses, symptoms, and procedures for claims processing. While this system is largely used …
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 the address that you specify. …
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 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), a lentiviral vector-based gene therapy …
In Memorial or Honorarium – October 2020
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 …
Newborn Screening Saves Lives Reauthorization
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 …