Zobrazeno 1 - 2
of 2
pro vyhledávání: '"neuropatías amiloideas"'
Autor:
Juan Buades, Hartmut Schmidt, Akshay Vaishnaw, Josep M. Campistol, David Adams, Jean Pouget, Jared Gollob, Brian Bettencourt, Ole B. Suhr, Isabel Conceição, Teresa Coelho, John L. Berk, Márcia Waddington-Cruz
Publikováno v:
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, BioMed Central, 2015, 10 (109 ), ⟨10.1186/s13023-015-0326-6⟩
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Agência para a Sociedade do Conhecimento (UMIC)-FCT-Sociedade da Informação
instacron:RCAAP
Orphanet Journal of Rare Diseases, BioMed Central, 2015, 10 (109 ), ⟨10.1186/s13023-015-0326-6⟩
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Agência para a Sociedade do Conhecimento (UMIC)-FCT-Sociedade da Informação
instacron:RCAAP
Background: Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d843bffbb5a94fb26b80776a45cbe3cb
http://europepmc.org/articles/PMC4559363
http://europepmc.org/articles/PMC4559363
Autor:
Juan Reinés, Tomás Vera, Mercedes Martín, Hernán Serra, Ma Margarita Campins, José Miguel Millán, Cristina Lezaun, Manuel Cruz
Publikováno v:
Orphanet Journal of Rare Diseases
Background: Transthyretin-associated Familial Amyloid Polyneuropathy (TTR-FAP) is an autosomal dominant disease caused by the deposition of abnormal transthyretin that results from a gene mutation. Although rare worldwide, there are descriptions of s
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::53788b9aa3275e51ad90304f81fd045f
http://europepmc.org/articles/PMC3941569
http://europepmc.org/articles/PMC3941569