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Therapeutic Advances in Rare Disease|August 5, 2026
First bone marrow transplantation for mucopolysaccharidosis type I in Vietnam: a case reportLe Nguyen-Ngoc-Quynh, Ngoc Can-Thi-Bich, Duong Dang-Anh, et al.Therapeutic Advances in Rare Disease|July 30, 2026
Treatment outcomes maintained in Hunter syndrome patients: a case series on switching from idursulfase to idursulfase beta in BelarusAnna KulpanovichTherapeutic Advances in Rare Disease|August 22, 2026
2000-2025: Emerging therapies in primary mitochondrial disease: A structured review, horizon scan, and roadmap for the futureAmel Karaa, Maria Isabel G Lopez Sanchez, Clare Stuart, et al.Therapeutic Advances in Rare Disease|September 7, 2026
Progress and perspectives in primary mitochondrial myopathy: Highlights from the 2025 mitochondrial medicine masterclass with a focus on TK2 deficiencyAustin Larson, Jared Goettemoeller, Ken Goettemoeller, et al.Therapeutic Advances in Rare Disease|September 8, 2026
Endothelial-mitochondrial coupling in mitochondrial disease: A systematic review and quantitative synthesis of vascular, biochemical, and oxidative bioenergetic dysfunctionJose A Adams, Tiffany Ko, Laith R Sultan, et al.Therapeutic Advances in Rare Disease|September 10, 2026
Evaluation of muscle microvascular perfusion in primary mitochondrial disease by contrast-enhanced ultrasound: Feasibility studyLaith R Sultan, Trudy A Morgan, Daniel E McGinn, et al.Pageof 13