Literature DB >> 31488615

Combination therapy with nusinersen and AVXS-101 in SMA type 1.

Bo Hoon Lee1, Erin Collins1, Leann Lewis1, Debra Guntrum1, Katy Eichinger1, Karen Voter1, Hoda Z Abdel-Hamid1, Emma Ciafaloni2.   

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Year:  2019        PMID： 31488615     DOI： 10.1212/WNL.0000000000008207

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


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  10 in total

1.  Batten disease: an expert update on agents in preclinical and clinical trials.

Authors:  Margaux C Masten; Jonathan W Mink; Erika F Augustine
Journal:  Expert Opin Investig Drugs       Date:  2020-11-01       Impact factor: 6.206

Review 2.  Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders.

Authors:  Flavien Bizot; Adeline Vulin; Aurélie Goyenvalle
Journal:  Drugs       Date:  2020-09       Impact factor: 9.546

Review 3.  Proteinopathies associated to repeat expansion disorders.

Authors:  Anthony Fourier; Isabelle Quadrio
Journal:  J Neural Transm (Vienna)       Date:  2022-01-24       Impact factor: 3.575

4.  Spinal Muscular Atrophy and Communicating Hydrocephalus: A Novel or a Well-Established Rare Association?

Authors:  Fatimah Z Alkhars; Nabil Almajhad; Jaafer Al-Obaid; Fatimah Alghadeer; Ahmed Y Bo Ali
Journal:  Cureus       Date:  2020-11-11

Review 5.  Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy.

Authors:  Tejal Aslesh; Toshifumi Yokota
Journal:  Cells       Date:  2022-01-26       Impact factor: 6.600

6.  Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.

Authors:  Arlene M D'Silva; Sandra Holland; Didu Kariyawasam; Karen Herbert; Peter Barclay; Anita Cairns; Suzanna C MacLennan; Monique M Ryan; Hugo Sampaio; Nicholas Smith; Ian R Woodcock; Eppie M Yiu; Ian E Alexander; Michelle A Farrar
Journal:  Ann Clin Transl Neurol       Date:  2022-02-16       Impact factor: 4.511

7.  Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients.

Authors:  Crystal J J Yeo; Zachary Simmons; Darryl C De Vivo; Basil T Darras
Journal:  Ann Neurol       Date:  2022-01-25       Impact factor: 11.274

8.  Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy.

Authors:  Leigh Ramos-Platt; Lauren Elman; Perry B Shieh
Journal:  Int J Gen Med       Date:  2022-09-17

9.  European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.

Authors:  Janbernd Kirschner; Nina Butoianu; Nathalie Goemans; Jana Haberlova; Anna Kostera-Pruszczyk; Eugenio Mercuri; W Ludo van der Pol; Susana Quijano-Roy; Thomas Sejersen; Eduardo F Tizzano; Andreas Ziegler; Laurent Servais; Francesco Muntoni
Journal:  Eur J Paediatr Neurol       Date:  2020-07-09       Impact factor: 3.140

Review 10.  New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?

Authors:  Tai-Heng Chen
Journal:  Int J Mol Sci       Date:  2020-05-07       Impact factor: 5.923
  10 in total

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