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First pharmacological option for early intervention
Original title: “Nezglyal approved in EU for childhood cALD”
European regulators move closer to approving option for young male patients
Original title: “Nezglyal receives positive CHMP opinion supporting treatment access for boys with cALD”
complex lipidome as a driver of tissue specific pathology in adrenoleukodystrophy
Original title: “The complex lipidome as a driver of tissue-specific pathology in adrenoleukodystrophy.”
profiling of fatty acids and lipids in animal and human tissues yields new leads for disease progression biomarkers of x linked adrenoleukodystrophy
Original title: “Profiling of fatty acids and lipids in animal and human tissues yields new leads for disease progression biomarkers of X-linked adrenoleukodystrophy.”
pediatric x linked adrenoleukodystrophy phenotypes variants and hsct outcomes
Original title: “Pediatric X-linked adrenoleukodystrophy: phenotypes, variants, and HSCT outcomes.”
Gene therapies cost $2.1M to $4.25M. Insurance barriers and Medicaid gaps are reshaping access for rare disease families. A single infusion for a child with this fatal neurodegenerative disease carries a price tag higher than most people will earn in a lifetime. Hemgenix, approved in 2022 for hemophilia B, costs $3.5 million. Lyfgenia, for sickle cell disease, is $3.1 million. Casgevy, the first CRISPR-based gene therapy by Vertex, is $2.2 million. Bluebird bio developed gene therapies for beta-thalassemia (Zynteglo), sickle cell disease (Lyfgenia), and cerebral adrenoleukodystrophy (Skysona). These were not failed therapies. They worked. Patients got better. NewsGene TherapyHealthcare AccessRare Disease PolicyMarch 29, 2026 · Gene therapies offer cures for previously untreatable rare diseases, but prices reaching $4.25 million create devastating access barriers. Therapies that literally cure genetic diseases once thought incurable are now available. But for the families who need them most, these breakthroughs have become locked behind a pricing wall so high it is often impenetrable. The gap between scientific innovation and patient access has never been wider. A therapy that works exists, yet most patients cannot access it. This is not a hypothetical problem. It is the daily reality for families navigating a rare disease diagnosis.
Original title: “Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend”
grey zone project risk based classification of abcd1 variants in x linked adrenoleukodystrophy
Original title: “The Grey Zone Project: Risk-Based Classification of ABCD1 Variants in X-Linked Adrenoleukodystrophy.”
adrenoleukodystrophy in adults phenotypic characterisation and natural history in a large cohort
Original title: “Adrenoleukodystrophy in adults: phenotypic characterisation and natural history in a large cohort.”
The first clinical application of lentiviral-based gene therapy was for treatment of inherited metabolic disorders including X-linked adrenoleukodystrophy (Eichler et al., 2017), metachromatic leukodystrophy (Rosenberg et al., 2016), and mucopolysaccharidoses (Kinsella et al., 2020), with the aim of increasing enzyme bioavailability and brain entry (Begley et al., 2008) for correction of the neuropathological phenotype (see below in the disease sections for more details).
Original title: “Frontiers | Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord”