ULF received donations in memorial or honorarium for the following during the months of May through July 2024. 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 Dan Mulligan Troy Glendenning Tara Nunez Quantum Crossing, LLC Master Built …
On August 1, 2024, Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three pediatric gene therapy programs. Through this transaction, Passage Bio has granted GEMMA Biotherapeutics the rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy. …
ULF received donations in memorial or honorarium for the following during the month of April 2024. 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 Brad & Lance Yotti James Bowersett Sue Bowersett Daxton Wallace Sandra Wallace Mia …
Learn more at www.cald-study.com!
ULF received donations in memorial or honorarium for the following during the month of March 2024. 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 BRAMBLEBOX TURTLE Melissa Kapoor Daxton Wallace Sandra Wallace Liz Thornton Kevin Dewhurst Mygelybelly …
The ULF is thrilled to share this press release from Orchard Therapeutics regarding their new FDA approved therapy for MLD! “Lenmeldy is truly a paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment, particularly when administered prior to the onset of symptoms,” said …
The ULF is shared to be able to share this press release from the Food & Drug Association: “Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).” …
FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy Read More »
ULF received donations in memorial or honorarium for the following during the month of February 2024. 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 Adele Moss Claudette Alice Collier Benjamin Rosenberg Ari Rosenberg live stream on Twitch.tv …
The ULF is happy to share this information on behalf of Vigil Neuro. Contact information is listed below for more information. Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP) A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia If you are interested in learning …
