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European Journal of Human Genetics : EJHG
|
June 7, 2007
A comprehensive screen for SNP associations on chromosome region 5q31-33 in Swedish/Norwegian celiac disease families
Silja Svanstrøm Amundsen, Svetlana Adamovic, Asa Hellqvist, et al.
Nature Reviews. Drug Discovery
|
December 15, 2019
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Nature Reviews. Drug Discovery
|
January 9, 2020
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Neuropsychobiology
|
May 15, 2009
DTNBP1, NRG1, DAOA, DAO and GRM3 polymorphisms and schizophrenia: an association study
Erik G Jönsson, Peter Saetre, Maria Vares, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Gene Therapy
|
March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
Frontiers in Medicine
|
September 13, 2021
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective
Maria E Sheean, Frauke Naumann-Winter, Giuseppe Capovilla, et al.
Drug Discovery Today
|
October 9, 2017
Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions
Maria E Sheean, Violeta Stoyanova-Beninska, Giuseppe Capovilla, et al.
Drug Discovery Today
|
April 29, 2020
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations
Rick A Vreman, Angela S de Ruijter, Anna Zawada, et al.
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of 5
Search research articles
Search
Showing results (31-40 of 41) with videos related to
Sort By:
Page
of 5
European Journal of Human Genetics : EJHG
|
June 7, 2007
A comprehensive screen for SNP associations on chromosome region 5q31-33 in Swedish/Norwegian celiac disease families
Silja Svanstrøm Amundsen, Svetlana Adamovic, Asa Hellqvist, et al.
Nature Reviews. Drug Discovery
|
December 15, 2019
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Nature Reviews. Drug Discovery
|
January 9, 2020
Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Erik Tambuyzer, Benjamin Vandendriessche, Christopher P Austin, et al.
Neuropsychobiology
|
May 15, 2009
DTNBP1, NRG1, DAOA, DAO and GRM3 polymorphisms and schizophrenia: an association study
Erik G Jönsson, Peter Saetre, Maria Vares, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Gene Therapy
|
March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
Frontiers in Medicine
|
September 13, 2021
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective
Maria E Sheean, Frauke Naumann-Winter, Giuseppe Capovilla, et al.
Drug Discovery Today
|
October 9, 2017
Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions
Maria E Sheean, Violeta Stoyanova-Beninska, Giuseppe Capovilla, et al.
Drug Discovery Today
|
April 29, 2020
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations
Rick A Vreman, Angela S de Ruijter, Anna Zawada, et al.
Page
of 5