Literature DB >> 29132728

Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience.

Sonia Messina1, Marika Pane2, Valeria Sansone3, Claudio Bruno4, Michela Catteruccia5, Giuseppe Vita1, Concetta Palermo2, Emilio Albamonte3, Marina Pedemonte4, Enrico Bertini6, Luca Binetti7, Eugenio Mercuri8.   

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Year:  2017        PMID： 29132728     DOI： 10.1016/j.nmd.2017.09.006

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  12 in total

Review 1.  Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle.

Authors:  Giuseppe Vita; Gian Luca Vita; Olimpia Musumeci; Carmelo Rodolico; Sonia Messina
Journal:  Neurol Sci       Date:  2019-02-25       Impact factor: 3.307

2.  Therapeutic oligonucleotides in cardiovascular and metabolic diseases: insights for the internist.

Authors:  Domenico Girelli; Fabiana Busti; Giacomo Marchi; Nicola Martinelli; Oliviero Olivieri
Journal:  Intern Emerg Med       Date:  2018-03-01       Impact factor: 3.397

Review 3.  Spinal muscular atrophy - insights and challenges in the treatment era.

Authors:  Eugenio Mercuri; Maria Carmela Pera; Mariacristina Scoto; Richard Finkel; Francesco Muntoni
Journal:  Nat Rev Neurol       Date:  2020-10-14       Impact factor: 42.937

4.  An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

Authors:  Jennifer M Kwon; Kapil Arya; Nancy Kuntz; Han C Phan; Cory Sieburg; Kathryn J Swoboda; Aravindhan Veerapandiyan; Beverly Assman; Silvia Bader-Weder; Travis L Dickendesher; Jennifer Hansen; Helen Lin; Ying Yan; Vamshi K Rao
Journal:  Ann Clin Transl Neurol       Date:  2022-05-14       Impact factor: 5.430

5.  Intrathecal administration of Nusinersen in type 1 SMA: successful psychological program in a single Italian center.

Authors:  Stefania La Foresta; Cristina Faraone; Maria Sframeli; Gian Luca Vita; Massimo Russo; Claudia Profazio; Immacolata Rulli; Eloisa Gitto; Antonio Versaci; Sonia Messina; Giuseppe Vita
Journal:  Neurol Sci       Date:  2018-07-25       Impact factor: 3.307

6.  An observational study of functional abilities in infants, children, and adults with type 1 SMA.

Authors:  Marika Pane; Concetta Palermo; Sonia Messina; Valeria A Sansone; Claudio Bruno; Michela Catteruccia; Maria Sframeli; Emilio Albamonte; Marina Pedemonte; Adele D'Amico; Giorgia Brigati; Roberto de Sanctis; Giorgia Coratti; Simona Lucibello; Enrico Bertini; Giuseppe Vita; Francesco Danilo Tiziano; Eugenio Mercuri
Journal:  Neurology       Date:  2018-07-25       Impact factor: 9.910

7.  Advances in Treatment of Spinal Muscular Atrophy - New Phenotypes, New Challenges, New Implications for Care.

Authors:  David C Schorling; Astrid Pechmann; Janbernd Kirschner
Journal:  J Neuromuscul Dis       Date:  2020

8.  Clinical Experience of Nusinersen in a Broad Spectrum of Spinal Muscular Atrophy: A Retrospective Study.

Authors:  Ae Ryoung Kim; Jong-Mok Lee; Yu-Sun Min; Hoseok Lee; Dongsub Kim; Su-Kyeong Hwang; Soonhak Kwon; Yun Jeong Lee
Journal:  Ann Indian Acad Neurol       Date:  2020-10-07       Impact factor: 1.383

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

10.  The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage.

Authors:  Anna Ambrosini; Daniela Calabrese; Francesco Maria Avato; Felice Catania; Guido Cavaletti; Maria Carmela Pera; Antonio Toscano; Giuseppe Vita; Lucia Monaco; Davide Pareyson
Journal:  Orphanet J Rare Dis       Date:  2018-10-04       Impact factor: 4.123
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