Literature DB >> 30045956

Precious SMA natural history data: A benchmark to measure future treatment successes.

Basil T Darras1, Darryl C De Vivo2.   

Abstract

Mesh：

Year:  2018        PMID： 30045956     DOI： 10.1212/WNL.0000000000006026

Source DB:  PubMed          Journal:  Neurology        ISSN： 0028-3878            Impact factor:   9.910


× No keyword cloud information.
  7 in total

1.  Nusinersen Treatment in Adults With Spinal Muscular Atrophy.

Authors:  Tina Duong; Connie Wolford; Michael P McDermott; Chelsea E Macpherson; Amy Pasternak; Allan M Glanzman; William B Martens; Elizabeth Kichula; Basil T Darras; Darryl C De Vivo; Zarazuela Zolkipli-Cunningham; Richard S Finkel; Michael Zeineh; Max Wintermark; Jacinda Sampson; Katharine A Hagerman; Sally Dunaway Young; John W Day
Journal:  Neurol Clin Pract       Date:  2021-06

Review 2.  Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence.

Authors:  Helgi Thor Hjartarson; Kristofer Nathorst-Böös; Thomas Sejersen
Journal:  Drug Des Devel Ther       Date:  2022-06-16       Impact factor: 4.319

Review 3.  Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy.

Authors:  Didu S T Kariyawasam; Arlene D'Silva; Cindy Lin; Monique M Ryan; Michelle A Farrar
Journal:  Front Neurol       Date:  2019-08-19       Impact factor: 4.003

4.  Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.

Authors:  Darryl C De Vivo; Enrico Bertini; Kathryn J Swoboda; Wuh-Liang Hwu; Thomas O Crawford; Richard S Finkel; Janbernd Kirschner; Nancy L Kuntz; Julie A Parsons; Monique M Ryan; Russell J Butterfield; Haluk Topaloglu; Tawfeg Ben-Omran; Valeria A Sansone; Yuh-Jyh Jong; Francy Shu; John F Staropoli; Douglas Kerr; Alfred W Sandrock; Christopher Stebbins; Marco Petrillo; Gabriel Braley; Kristina Johnson; Richard Foster; Sarah Gheuens; Ishir Bhan; Sandra P Reyna; Stephanie Fradette; Wildon Farwell
Journal:  Neuromuscul Disord       Date:  2019-09-12       Impact factor: 4.296

5.  Natural history of lung function in spinal muscular atrophy.

Authors:  Camiel A Wijngaarde; Esther S Veldhoen; Ruben P A van Eijk; Marloes Stam; Louise A M Otto; Fay-Lynn Asselman; Roelie M Wösten-van Asperen; Erik H J Hulzebos; Laura P Verweij-van den Oudenrijn; Bart Bartels; Inge Cuppen; Renske I Wadman; Leonard H van den Berg; Cornelis K van der Ent; W Ludo van der Pol
Journal:  Orphanet J Rare Dis       Date:  2020-04-10       Impact factor: 4.123

6.  Natural history of respiratory muscle strength in spinal muscular atrophy: a prospective national cohort study.

Authors:  Esther S Veldhoen; Camiel A Wijngaarde; Erik H J Hulzebos; Roelie M Wösten-van Asperen; Renske I Wadman; Ruben P A van Eijk; Fay Lynn Asselman; Marloes Stam; Louise A M Otto; Inge Cuppen; Feline E V Scheijmans; Laura P Verweij-van den Oudenrijn; Bart Bartels; Michael A Gaytant; Cornelis K van der Ent; W Ludo van der Pol
Journal:  Orphanet J Rare Dis       Date:  2022-02-21       Impact factor: 4.123

Review 7.  In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy.

Authors:  Kristine S Ojala; Emily J Reedich; Christine J DiDonato; Stephen D Meriney
Journal:  Brain Sci       Date:  2021-02-05
  7 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.