Year: 2025

28th ASGCT Annual Meeting- Empowering Patients 2025: A Cell and Gene Therapies Summit

News / February 19, 2025

Hosted by American Society of Gene + Cell Therapy This inaugural virtual event March 12 and 13, 2025 (9 a.m. – 12:30 p.m. ET on both days), will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates, patients, and caregivers who are …

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In Memorial and Honor: January 2025

In Memory or In Honor Donation / February 18, 2025

ULF received donations in memorial or honorarium for the following during the month of January 2025. 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 Bob Hodge Jr., Brian Greene, Garner Hicks, and Tom Cunningham Sallie Griffiths Daxton …

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In Memorial and Honor: October – December 2024

In Memory or In Honor Donation / January 24, 2025

ULF received donations in memorial or honorarium for the following during the months of October through December 2024. 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 Adam Sledzik Stanislaw Sledzik CTX is very bad ,rare disease. Adrianna …

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Study: Progression of Spinal Cord Disease in Adult Men With ALD

Research / January 22, 2025

The article, Progression of Spinal Cord Disease in Adult Men with Adrenoleukodystrophy, details the results of a long-term study on the progression of spinal cord disease in adult men with ALD. The study, which followed 79 patients over seven years, used clinical tools like the EDSS, SSPROM, and 6MWT to measure disease progression. It showed …

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Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile MLD: a long-term follow-up pilot study

Research / January 22, 2025

The study “Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study” investigates a pioneering treatment for juvenile metachromatic leukodystrophy (MLD), a rare, inherited neurological disorder caused by a deficiency in the ARSA enzyme. This enzyme deficiency leads to the toxic accumulation of sulfatides, causing progressive demyelination (damage …

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