Publications by authors named "Marc Dooms"

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Lessons learned from the RE(ACT) conference on medical devices for rare diseases.
Anneliene H Jonker, Tim Buckinx, Lucia Pannese, Paulien Klap, José-Alain Sahel, Marc Dooms

Eur J Med Genet· December 2024

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Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.
Szczepan Jakubowski, Przemysław Holko, Rafał Nowak, Marisa Warmuth, Marc Dooms

Front Pharmacol· November 2024

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Orphan and paediatric medical devices in Europe: recommendations to support their availability for on-label and off-label clinical indications.
Tom Melvin, Marc M Dooms, Berthold Koletzko, Mark A Turner, Damien Kenny

Expert Rev Med Devices· October 2024

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Global health for rare diseases through primary care.
Gareth Baynam, Adam L Hartman, Mary Catherine V Letinturier, Matt Bolz-Johnson, Prescilla Carrion, Marc Dooms

Lancet Glob Health· July 2024

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Targeting shared molecular etiologies to accelerate drug development for rare diseases.
Galliano Zanello, Macarena Garrido-Estepa, Ana Crespo, Daniel O'Connor, Rima Nabbout, Marc Dooms

EMBO Mol Med· July 2023

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Editorial: Prevention, diagnosis and treatment of rare disorders.
Timothy M Cox, Anna Tylki-Szymańska, Ségolène Aymé, Marc Dooms

Front Pharmacol· September 2022

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Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group).
Annemieke Aartsma-Rus, Marc Dooms, Yann Le Cam

Front Pharmacol· December 2021

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Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.
Sweta Shah, Marc Marie Dooms, Sofia Amaral-Garcia, Mariana Igoillo-Esteve

Front Pharmacol· December 2021

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A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe.
Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens

Front Pharmacol· November 2021

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Clinical Research in Neonates: Redesigning the Informed Consent Process in the Digital Era.
Evelien De Sutter, Birte Coopmans, Femke Vanendert, Marc Dooms, Karel Allegaert

Front Pediatr· September 2021

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Orphan Drugs, Compounded Medication and Pharmaceutical Commons.
Kim Hendrickx, Marc Dooms

Front Pharmacol· September 2021

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Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.
Ewa Stawowczyk, Krzysztof Piotr Malinowski, Paweł Kawalec, Rafał Bobiński, Jacek Siwiec, Marc Dooms

Front Pharmacol· November 2019

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Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium.
Liese Vandeborne, Eline van Overbeeke, Marc Dooms, Birgit De Beleyr, Isabelle Huys

Orphanet J Rare Dis· May 2019

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Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice.
Rosanne Janssens, Eline van Overbeeke, Lotte Verswijvel, Lissa Meeusen, Carolien Coenegrachts, Marc Dooms

Front Med (Lausanne)· October 2018

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Off-label use of medicines: The need for good practice guidelines.
Marc Dooms, James Killick

Int J Risk Saf Med· June 2018

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Compounded medication for patients with rare diseases.
Marc Dooms, Maria Carvalho

Orphanet J Rare Dis· January 2018

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Noninvasive prenatal testing: a survey of young (future) parents in Flanders.
Pascal Borry, Maddalena Favaretto, Ann Batthyany, Eline Boey, Sophie Van Tongerloo, Marc Dooms

Per Med· January 2018

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From promising molecules to orphan drugs: Early clinical drug development.
Marc Dooms

Intractable Rare Dis Res· February 2017

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Off-label use of orphan medicinal products: a Belgian qualitative study.
Marc Dooms, David Cassiman, Steven Simoens

Orphanet J Rare Dis· October 2016

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Rembert Dodoens (1517-1585). Pioneer in Rare Diseases and Orphan Drugs.
Marc Dooms

Bull Cercle Benelux Hist Pharm· March 2016

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Rare diseases and orphan drugs: 500 years ago.
Marc M Dooms

Orphanet J Rare Dis· December 2015

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[Medication: 15 years 'EU-directive orphan drugs'].
Marc Dooms

Bull Cercle Benelux Hist Pharm· March 2015

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Cost-effectiveness assessment of orphan drugs: a scientific and political conundrum.
Steven Simoens, Eline Picavet, Marc Dooms, David Cassiman, Thomas Morel

Appl Health Econ Health Policy· February 2013

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Orphan drugs for rare diseases: is it time to revisit their special market access status?
Steven Simoens, David Cassiman, Marc Dooms, Eline Picavet

Drugs· July 2012

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