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Health Research Policy and Systems
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October 17, 2025
Integrative research and innovation strategy for rare diseases. Insights from the 5-year European joint programme on rare diseases, including analysis to inform recommendations for future actions
María Del Carmen Sánchez-González, Rodrigo Sarmiento-Suárez, Laura Lee Cellai, et al.
Journal of the Neurological Sciences
|
July 6, 2015
Protracted late infantile ceroid lipofuscinosis due to TPP1 mutations: Clinical, molecular and biochemical characterization in three sibs
Raffaella Di Giacopo, Luciano Cianetti, Viviana Caputo, et al.
American Journal of Human Genetics
|
December 29, 2005
NF1 gene mutations represent the major molecular event underlying neurofibromatosis-Noonan syndrome
Alessandro De Luca, Irene Bottillo, Anna Sarkozy, et al.
American Journal of Human Genetics
|
January 17, 2012
A restricted spectrum of mutations in the SMAD4 tumor-suppressor gene underlies Myhre syndrome
Viviana Caputo, Luciano Cianetti, Marcello Niceta, et al.
Cancer Genetics and Cytogenetics
|
April 25, 2006
Activating PTPN11 mutations play a minor role in pediatric and adult solid tumors
Simone Martinelli, Claudio Carta, Elisabetta Flex, et al.
Human Mutation
|
October 21, 2006
Diversity, parental germline origin, and phenotypic spectrum of de novo HRAS missense changes in Costello syndrome
Giuseppe Zampino, Francesca Pantaleoni, Claudio Carta, et al.
Journal of Neuromuscular Diseases
|
October 1, 2025
The FAIR journey of a patient-driven registry: Reflections and practical solutions from the Duchenne Data Platform FAIRification experience
Nawel Lalout, Mark D Wilkinson, Dagmar Wandrei, et al.
Frontiers in Molecular Biosciences
|
May 26, 2023
Resources and tools for rare disease variant interpretation
Luana Licata, Allegra Via, Paola Turina, et al.
European Journal of Human Genetics : EJHG
|
February 4, 2018
The RD-Connect Registry & Biobank Finder: a tool for sharing aggregated data and metadata among rare disease researchers
Sabina Gainotti, Paola Torreri, Chiuhui Mary Wang, et al.
Biomed Research International
|
December 8, 2017
Linked Registries: Connecting Rare Diseases Patient Registries through a Semantic Web Layer
Pedro Sernadela, Lorena González-Castro, Claudio Carta, et al.
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Search research articles
Search
Showing results (21-30 of 39) with videos related to
Sort By:
Page
of 4
Health Research Policy and Systems
|
October 17, 2025
Integrative research and innovation strategy for rare diseases. Insights from the 5-year European joint programme on rare diseases, including analysis to inform recommendations for future actions
María Del Carmen Sánchez-González, Rodrigo Sarmiento-Suárez, Laura Lee Cellai, et al.
Journal of the Neurological Sciences
|
July 6, 2015
Protracted late infantile ceroid lipofuscinosis due to TPP1 mutations: Clinical, molecular and biochemical characterization in three sibs
Raffaella Di Giacopo, Luciano Cianetti, Viviana Caputo, et al.
American Journal of Human Genetics
|
December 29, 2005
NF1 gene mutations represent the major molecular event underlying neurofibromatosis-Noonan syndrome
Alessandro De Luca, Irene Bottillo, Anna Sarkozy, et al.
American Journal of Human Genetics
|
January 17, 2012
A restricted spectrum of mutations in the SMAD4 tumor-suppressor gene underlies Myhre syndrome
Viviana Caputo, Luciano Cianetti, Marcello Niceta, et al.
Cancer Genetics and Cytogenetics
|
April 25, 2006
Activating PTPN11 mutations play a minor role in pediatric and adult solid tumors
Simone Martinelli, Claudio Carta, Elisabetta Flex, et al.
Human Mutation
|
October 21, 2006
Diversity, parental germline origin, and phenotypic spectrum of de novo HRAS missense changes in Costello syndrome
Giuseppe Zampino, Francesca Pantaleoni, Claudio Carta, et al.
Journal of Neuromuscular Diseases
|
October 1, 2025
The FAIR journey of a patient-driven registry: Reflections and practical solutions from the Duchenne Data Platform FAIRification experience
Nawel Lalout, Mark D Wilkinson, Dagmar Wandrei, et al.
Frontiers in Molecular Biosciences
|
May 26, 2023
Resources and tools for rare disease variant interpretation
Luana Licata, Allegra Via, Paola Turina, et al.
European Journal of Human Genetics : EJHG
|
February 4, 2018
The RD-Connect Registry & Biobank Finder: a tool for sharing aggregated data and metadata among rare disease researchers
Sabina Gainotti, Paola Torreri, Chiuhui Mary Wang, et al.
Biomed Research International
|
December 8, 2017
Linked Registries: Connecting Rare Diseases Patient Registries through a Semantic Web Layer
Pedro Sernadela, Lorena González-Castro, Claudio Carta, et al.
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of 4