Literature DB >> 27444223

Patients as key partners in rare disease drug development.

Max G Bronstein1, Emil D Kakkis2,3.   

Abstract

Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit-risk profile of potential therapies.

Entities:  

Mesh：

Year:  2016        PMID： 27444223     DOI： 10.1038/nrd.2016.133

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


  8 in total

Review 1.  From promising molecules to orphan drugs: Early clinical drug development.

Authors:  Marc Dooms
Journal:  Intractable Rare Dis Res       Date:  2017-02

Review 2.  Clinical Trial Design for Disease-Modifying Therapies for Genetic Epilepsies.

Authors:  Dylan C Brock; Scott Demarest; Tim A Benke
Journal:  Neurotherapeutics       Date:  2021-09-30       Impact factor: 6.088

3.  Role of Patients and Parents in Pediatric Drug Development.

Authors:  Vivian W L Tsang; Leanne West; Christine Woods; Chester J Koh; Susan McCune; Theresa Mullin; Sharon R Smith; Segolene Gaillard; Joana Claverol; Begonya Nafria; Jennifer Preston; Pamela Dicks; Charles Thompson
Journal:  Ther Innov Regul Sci       Date:  2019-01-20       Impact factor: 1.778

4.  Dynamic Consent: a potential solution to some of the challenges of modern biomedical research.

Authors:  Isabelle Budin-Ljøsne; Harriet J A Teare; Jane Kaye; Stephan Beck; Heidi Beate Bentzen; Luciana Caenazzo; Clive Collett; Flavio D'Abramo; Heike Felzmann; Teresa Finlay; Muhammad Kassim Javaid; Erica Jones; Višnja Katić; Amy Simpson; Deborah Mascalzoni
Journal:  BMC Med Ethics       Date:  2017-01-25       Impact factor: 2.652

5.  Developing and evaluating rare disease educational materials co-created by expert clinicians and patients: the paradigm of congenital hypogonadotropic hypogonadism.

Authors:  Corin Badiu; Marco Bonomi; Ivan Borshchevsky; Martine Cools; Margarita Craen; Cristina Ghervan; Michael Hauschild; Eli Hershkovitz; Erik Hrabovszky; Anders Juul; Soo-Hyun Kim; Phillip Kumanov; Beatriz Lecumberri; Manuel C Lemos; Vassos Neocleous; Marek Niedziela; Sandra Pekic Djurdjevic; Luca Persani; Franziska Phan-Hug; Duarte Pignatelli; Nelly Pitteloud; Vera Popovic; Richard Quinton; Nicos Skordis; Neil Smith; Magdalena Avbelj Stefanija; Cheng Xu; Jacques Young; Andrew A Dwyer
Journal:  Orphanet J Rare Dis       Date:  2017-03-20       Impact factor: 4.123

Review 6.  Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures.

Authors:  Thomas Morel; Stefan J Cano
Journal:  Orphanet J Rare Dis       Date:  2017-11-02       Impact factor: 4.123

7.  Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy.

Authors:  F Eichler; Caroline Sevin; M Barth; F Pang; K Howie; M Walz; A Wilds; C Calcagni; C Chanson; L Campbell
Journal:  Orphanet J Rare Dis       Date:  2022-10-04       Impact factor: 4.303

Review 8.  How Surrogate and Chemical Genetics in Model Organisms Can Suggest Therapies for Human Genetic Diseases.

Authors:  Katherine A Strynatka; Michelle C Gurrola-Gal; Jason N Berman; Christopher R McMaster
Journal:  Genetics       Date:  2018-03       Impact factor: 4.562
  8 in total

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