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Therapeutic Advances in Rare Disease|May 14, 2023
Mannose treatment improves immune deficiency in mannose phosphate isomerase-congenital disorder of glycosylation: case report and review of literatureDiederik De Graef, Jehan Mousa, Marta Biderman Waberski, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Therapeutic angiogenesis in Buerger's disease: reviewing the treatment landscapeAntoine J Ribieras, Yulexi Y Ortiz, Zhao-Jun Liu, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Therapeutic and diagnostic advances in Stickler syndromeMartin Snead, Howard Martin, Peter Bale, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Conservative management of a fourth ventricular epidermoid in a patient with Gardner syndromeGordon D HellerTherapeutic Advances in Rare Disease|May 14, 2023
Role of red free imaging, retinal reflectance and fundus autofluorescence in Bietti crystalline dystrophy: case reportAbhidnya Surve, Akshaya Balaji, Shorya Vardhan Azad, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Marfan syndrome and the eye clinic: from diagnosis to managementHaseeb Akram, Jose Antonio Aragon-Martin, Aman ChandraTherapeutic Advances in Rare Disease|May 14, 2023
New avenues for therapy in mitochondrial optic neuropathiesWing Sum Vincent Ng, Matthieu Trigano, Thomas Freeman, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Improving patient informed consent for haemophilia gene therapy: the case for changeLaurence Woollard, Richard Gorman, Dakota J RosenfeltTherapeutic Advances in Rare Disease|March 6, 2024
A United States-based patient-reported adult polyglucosan body disease registry: initial resultsJacy Sparks, Francesco Michelassi, John L P Thompson, et al.Therapeutic Advances in Rare Disease|March 7, 2024
An integrated action plan to fund and support drug development for Dup15q syndrome: a patient organization perspectiveRyan Rogers-Hammond, Carrie HowellPageof 13