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Indicators Of Metachromatic Leukodystrophy
Metachromatic leukodystrophy is a genetic condition that occurs due to an enzyme deficiency. Patients with this disorder have ...
Orchard Therapeutics Announces Multiple Data Presentations and Receives 2025 New Treatment Award at the 21st Annual WORLDSymposium™
Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...
How a Virginia father’s simple act of generosity is honoring late son’s life
After Paul Jasman died from a rare disease last summer, his father began donating medical items to a Fredericksburg program ...
Jasman family's loss transforms into hope for others through medical device donations
After Paul Jasman died from a rare disease last summer, his father began donating medical items to a Fredericksburg program ...
Nature2y
Mutant NDUFV1 subunit of mitochondrial complex I causes leukodystrophy and myoclonic epilepsy
The clinical history of patients II:1 and II:2 (family A) is similar in course, clinical and biochemical findings: pregnancy and delivery were uneventful, and birth weight was normal. At the age ...
Release Therapeutics Announces New CEO To Advance Groundbreaking Treatment For Brain Disorders
Julien Grogg, PhD, MBA - CEO of Release Therapeutics Release Tx logo Dr. Julien Grogg will Lead the Company's Transition to Clinical ...