Last Updated: 09/26/2026
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Found 7 publications
Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series.
Journal: Orphanet journal of rare diseases
Published: March 30, 2018
Estimating the global prevalence of transthyretin familial amyloid polyneuropathy.
Journal: Muscle & nerve
Published: December 02, 2017
Global epidemiology of transthyretin hereditary amyloid polyneuropathy: a systematic review.
Journal: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
Published: April 25, 2017
Epidemiological and clinical characteristics of persons with transthyretin hereditary amyloid polyneuropathy: a global synthesis of 532 cases.
Journal: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
Published: April 25, 2017
Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships.
Journal: Orphanet journal of rare diseases
Published: May 26, 2015
Airway-related symptoms and surgeries in patients with mucopolysaccharidosis I.
Journal: The Annals of otology, rhinology, and laryngology
Published: September 13, 2014
The natural history of MPS I: global perspectives from the MPS I Registry.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: December 17, 2013
Showing 1-7 of 7
Last Updated: 09/26/2026