Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

doi:10.1016/S1474-4422(22)00339-8

Randomized Controlled Trial

. 2022 Dec;21(12):1110-1119.

doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14.

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson¹, Maria Mazurkiewicz-Bełdzińska², Kristy Rose³, Laurent Servais⁴, Hui Xiong⁵, Edmar Zanoteli⁶, Giovanni Baranello⁷, Claudio Bruno⁸, John W Day⁹, Nicolas Deconinck¹⁰, Andrea Klein¹¹, Eugenio Mercuri¹², Dmitry Vlodavets¹³, Yi Wang¹⁴, Angela Dodman¹⁵, Muna El-Khairi¹⁶, Ksenija Gorni¹⁷, Birgit Jaber¹⁸, Heidemarie Kletzl¹⁹, Eleni Gaki¹⁶, Paulo Fontoura¹⁷, Basil T Darras²⁰; FIREFISH Study Group

Collaborators, Affiliations

Collaborators

FIREFISH Study Group:
Joseph J Volpe, John Posner, Ulrich Kellner, Rosaline Quinlivan, Marianne Gerber, Omar Khwaja, Renata S Scalco, Timothy Seabrook, Armin Koch, Irina Balikova, Inge Joniau, Geraldine Accou, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Rodrigo de Holanda Mendonça, Ciro Matsui Jr, Ana Letícia Fornazieri Darcie, Cleide Machado, Maria Kiyoko Oyamada, Joyce Martini, Graziela Polido, Juliana Rodrigues Iannicelli, Juliana Caires de Oliveira Achili Ferreira, Chaoping Hu, Xiaomei Zhu, Chen Qian, Li Shen, Hui Li, Yiyun Shi, Shuizhen Zhou, Ying Xiao, Zhenxuan Zhou, Sujuan Wang, Tian Sang, Cuijie Wei, Hui Dong, Yiwen Cao, Jing Wen, Wenzhu Li, Lun Qin, Nina Barisic, Ivan Celovec, Martina Galiot Delic, Petra Kristina Ivkic, Nenad Vukojevic, Ivana Kern, Boris Najdanovic, Marin Skugor, Josipa Tomas, Odile Boespflug-Tanguy, Silvana De Lucia, Andrea Seferian, Emmanuel Barreau, Nabila Mnafek, Helene Peche, Allison Grange, Diem Trang Nguyen, Darko Milascevic, Shotaro Tachibana, Emanuela Pagliano, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, Marta Vigano, Claudia Dosi, Riccardo Zanin, Veronica Schembri, Noemi Brolatti, Giuseppe Rao, Elisa Tassara, Simone Morando, Paola Tacchetti, Marina Pedemonte, Enrico Priolo, Lorenza Sposetti, Giacomo Pietro Comi, Alessandra Govoni, Silvia Gabriella Osnaghi, Valeria Minorini, Francesca Abbati, Federica Fassini, Michaela Foa, Amalia Lopopolo, Marika Pane, Concetta Palermo, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, Guglielmo D'Amico, Lorenzo Orazi, Giorgia Coratti, Daniela Leone, Antonaci Laura, Roberto De Sanctis, Beatrice Berti, Naoki Kimura, Yasuhiro Takeshima, Hideki Shimomura, Tomoko Lee, Fumi Gomi, Takanobu Morimatsu, Toru Furukawa, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Sandra Modrzejewska, Anna Lemska, Evgenia Melnik, Svetlana Artemyeva, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Elena Litvinova, Cornelia Enzmann, Elea Galiart, Konstantin Gugleta, Christine Wondrusch Haschke, Haluk Topaloglu, Ibrahim Oncel, Nesibe Eroglu Ertugrul, Bahadir Konuskan, Bora Eldem, Sibel Kadayifçilar, Ipek Alemdaroglu, Seher Sari, Neslihan Bilgin, Aynur Ayse Karaduman, Fatma Gokcem Yildiz Sarikaya, Robert J Graham, Partha Ghosh, David Casavant, Alexis Levine, Rachael Titus, Amanda Engelbrekt, Lucia Ambrosio, Anne Fulton, Anna Maria Baglieri, Courtney Dias, Elizabeth Maczek, Amy Pasternak, Shannon Beres, Tina Duong, Richard Gee, Sally Young

Affiliations

¹ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy. Electronic address: riccardo.masson@istituto-besta.it.
² Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland.
³ Discipline of Physiotherapy, Faculty of Medicine and Health, University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW, Australia.
⁴ MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, Neuromuscular Center, CHU and University of Liège, Liège, Belgium; I-Motion - Hôpital Armand Trousseau, Paris, France.
⁵ Department of Pediatrics, Peking University First Hospital, Beijing, China.
⁶ Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brazil.
⁷ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, London, UK.
⁸ Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, University of Genoa, Genoa, Italy.
⁹ Department of Neurology, Stanford University, Palo Alto, CA, USA.
¹⁰ Neuromuscular Reference Center, UZ Gent, Ghent, Belgium; Neuromuscular Reference Center and Paediatric Neurology Department, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, Brussels, Belgium.
¹¹ Paediatric Neurology, University Children's Hospital Basel, Basel, Switzerland; Division of Neuropaediatrics, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.
¹² Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
¹³ Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, Moscow, Russia.
¹⁴ Department of Neurology, Children's Hospital of Fudan University, Shanghai, China.
¹⁵ Pharma Development Neurology, F Hoffmann-La Roche, Basel, Switzerland.
¹⁶ Roche Products, Welwyn Garden City, UK.
¹⁷ Product Development Medical Affairs-Neuroscience and Rare Disease, F Hoffmann-La Roche, Basel, Switzerland.
¹⁸ Pharma Development, Safety Risk Management, F Hoffmann-La Roche, Basel, Switzerland.
¹⁹ Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
²⁰ Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

PMID: 36244364
DOI: 10.1016/S1474-4422(22)00339-8

Randomized Controlled Trial

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson et al. Lancet Neurol. 2022 Dec.

. 2022 Dec;21(12):1110-1119.

doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14.

Authors

Collaborators

FIREFISH Study Group:
Joseph J Volpe, John Posner, Ulrich Kellner, Rosaline Quinlivan, Marianne Gerber, Omar Khwaja, Renata S Scalco, Timothy Seabrook, Armin Koch, Irina Balikova, Inge Joniau, Geraldine Accou, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Rodrigo de Holanda Mendonça, Ciro Matsui Jr, Ana Letícia Fornazieri Darcie, Cleide Machado, Maria Kiyoko Oyamada, Joyce Martini, Graziela Polido, Juliana Rodrigues Iannicelli, Juliana Caires de Oliveira Achili Ferreira, Chaoping Hu, Xiaomei Zhu, Chen Qian, Li Shen, Hui Li, Yiyun Shi, Shuizhen Zhou, Ying Xiao, Zhenxuan Zhou, Sujuan Wang, Tian Sang, Cuijie Wei, Hui Dong, Yiwen Cao, Jing Wen, Wenzhu Li, Lun Qin, Nina Barisic, Ivan Celovec, Martina Galiot Delic, Petra Kristina Ivkic, Nenad Vukojevic, Ivana Kern, Boris Najdanovic, Marin Skugor, Josipa Tomas, Odile Boespflug-Tanguy, Silvana De Lucia, Andrea Seferian, Emmanuel Barreau, Nabila Mnafek, Helene Peche, Allison Grange, Diem Trang Nguyen, Darko Milascevic, Shotaro Tachibana, Emanuela Pagliano, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, Marta Vigano, Claudia Dosi, Riccardo Zanin, Veronica Schembri, Noemi Brolatti, Giuseppe Rao, Elisa Tassara, Simone Morando, Paola Tacchetti, Marina Pedemonte, Enrico Priolo, Lorenza Sposetti, Giacomo Pietro Comi, Alessandra Govoni, Silvia Gabriella Osnaghi, Valeria Minorini, Francesca Abbati, Federica Fassini, Michaela Foa, Amalia Lopopolo, Marika Pane, Concetta Palermo, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, Guglielmo D'Amico, Lorenzo Orazi, Giorgia Coratti, Daniela Leone, Antonaci Laura, Roberto De Sanctis, Beatrice Berti, Naoki Kimura, Yasuhiro Takeshima, Hideki Shimomura, Tomoko Lee, Fumi Gomi, Takanobu Morimatsu, Toru Furukawa, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Sandra Modrzejewska, Anna Lemska, Evgenia Melnik, Svetlana Artemyeva, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Elena Litvinova, Cornelia Enzmann, Elea Galiart, Konstantin Gugleta, Christine Wondrusch Haschke, Haluk Topaloglu, Ibrahim Oncel, Nesibe Eroglu Ertugrul, Bahadir Konuskan, Bora Eldem, Sibel Kadayifçilar, Ipek Alemdaroglu, Seher Sari, Neslihan Bilgin, Aynur Ayse Karaduman, Fatma Gokcem Yildiz Sarikaya, Robert J Graham, Partha Ghosh, David Casavant, Alexis Levine, Rachael Titus, Amanda Engelbrekt, Lucia Ambrosio, Anne Fulton, Anna Maria Baglieri, Courtney Dias, Elizabeth Maczek, Amy Pasternak, Shannon Beres, Tina Duong, Richard Gee, Sally Young

Affiliations

¹ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy. Electronic address: riccardo.masson@istituto-besta.it.
² Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland.
³ Discipline of Physiotherapy, Faculty of Medicine and Health, University of Sydney and Sydney Children's Hospitals Network, Sydney, NSW, Australia.
⁴ MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, Neuromuscular Center, CHU and University of Liège, Liège, Belgium; I-Motion - Hôpital Armand Trousseau, Paris, France.
⁵ Department of Pediatrics, Peking University First Hospital, Beijing, China.
⁶ Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brazil.
⁷ Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital Trust, London, UK.
⁸ Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy; Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health - DINOGMI, University of Genoa, Genoa, Italy.
⁹ Department of Neurology, Stanford University, Palo Alto, CA, USA.
¹⁰ Neuromuscular Reference Center, UZ Gent, Ghent, Belgium; Neuromuscular Reference Center and Paediatric Neurology Department, Queen Fabiola Children's University Hospital, Université Libre de Bruxelles, Brussels, Belgium.
¹¹ Paediatric Neurology, University Children's Hospital Basel, Basel, Switzerland; Division of Neuropaediatrics, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.
¹² Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
¹³ Russian Children Neuromuscular Center, Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, Moscow, Russia.
¹⁴ Department of Neurology, Children's Hospital of Fudan University, Shanghai, China.
¹⁵ Pharma Development Neurology, F Hoffmann-La Roche, Basel, Switzerland.
¹⁶ Roche Products, Welwyn Garden City, UK.
¹⁷ Product Development Medical Affairs-Neuroscience and Rare Disease, F Hoffmann-La Roche, Basel, Switzerland.
¹⁸ Pharma Development, Safety Risk Management, F Hoffmann-La Roche, Basel, Switzerland.
¹⁹ Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
²⁰ Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

PMID: 36244364
DOI: 10.1016/S1474-4422(22)00339-8

Abstract

Background: Risdiplam is an orally administered therapy that modifies pre-mRNA splicing of the survival of motor neuron 2 (SMN2) gene and is approved for the treatment of spinal muscular atrophy. The FIREFISH study is investigating the safety and efficacy of risdiplam in treated infants with type 1 spinal muscular atrophy versus historical controls. The primary endpoint of part 2 of the FIREFISH study showed that infants with type 1 spinal muscular atrophy attained the ability to sit without support for at least 5 s after 12 months of treatment. Here, we report on the safety and efficacy of risdiplam in FIREFISH part 2 over 24 months of treatment.

Methods: FIREFISH is an ongoing, multicentre, open-label, two-part study. In FIREFISH part 2, eligible infants (aged 1-7 months at enrolment, with a genetically confirmed diagnosis of spinal muscular atrophy, and two SMN2 gene copies) were enrolled in 14 hospitals in ten countries across Europe, North America, South America, and Asia. Risdiplam was orally administered once daily at 0·2 mg/kg for infants between 5 months and 2 years of age; once an infant reached 2 years of age, the dose was increased to 0·25 mg/kg. Infants younger than 5 months started at 0·04 mg/kg (infants between 1 month and 3 months old) or 0·08 mg/kg (infants between 3 months and 5 months old), and this starting dose was adjusted to 0·2 mg/kg once pharmacokinetic data were available for each infant. The primary and secondary endpoints included in the statistical hierarchy and assessed at month 12 have been reported previously. Here we present the remainder of the secondary efficacy endpoints that were included in the statistical hierarchy at month 24: the ability to sit without support for at least 30 s, to stand alone, and to walk alone, as assessed by the Bayley Scales of Infant and Toddler Development, third edition gross motor subscale. These three endpoints were compared with a performance criterion of 5% that was defined based on the natural history of type 1 spinal muscular atrophy; the results were considered statistically significant if the lower limit of the two-sided 90% CI was above the 5% threshold. FIREFISH is registered with ClinicalTrials.gov, NCT02913482. Recruitment is closed; the 36-month extension period of the study is ongoing.

Findings: Between March 13 and Nov 19, 2018, 41 infants were enrolled in FIREFISH part 2. After 24 months of treatment, 38 infants were ongoing in the study and 18 infants (44% [90% CI 31-58]) were able to sit without support for at least 30 s (p<0·0001 compared with the performance criterion derived from the natural history of untreated infants with type 1 spinal muscular atrophy). No infants could stand alone (0 [90% CI 0-7]) or walk alone (0 [0-7]) after 24 months of treatment. The most frequently reported adverse event was upper respiratory tract infection, in 22 infants (54%); the most common serious adverse events were pneumonia in 16 infants (39%) and respiratory distress in three infants (7%).

Interpretation: Treatment with risdiplam over 24 months resulted in continual improvements in motor function and achievement of developmental motor milestones. The FIREFISH open-label extension phase will provide additional evidence regarding long-term safety and efficacy of risdiplam.

Funding: F Hoffmann-La Roche.

PubMed Disclaimer

Conflict of interest statement

Declaration of interests RM has received consulting fees from Biogen, F Hoffmann-La Roche, and Novartis Gene Therapies, and speaker honoraria from F Hoffmann-La Roche and Novartis Gene Therapies. RM reports that he received travel or meeting attendance support and fees for serving on advisory boards from F Hoffmann-La Roche, Novartis Gene Therapies, and Biogen. MMB has received speaker honoraria and payment for serving on advisory boards for F Hoffmann-La Roche, Sanofi, Novartis, Biogen, and UCB. KR reports she has received consulting fees from F Hoffmann-La Roche, Biogen, and Novartis; she has received support from Biogen for attending meetings; she has received speaker honoraria from F Hoffmann-La Roche and Biogen, and has served on advisory boards for F Hoffmann-La Roche and Biogen. LS reports grants from F Hoffmann-La Roche, Biogen, and Novartis, and consultancy fees from F Hoffmann-La Roche, Biogen, Novartis, BioHaven Pharmaceuticals, and Scholar Rock. He has received speaker fees from and served on advisory boards for F Hoffmann-La Roche, Biogen, and Novartis. He reports he holds the position of secretary at the World Muscle Society. HX declared no competing interests. EZ reports grants from Fundação de Amparo à Pesquisa do Estado de São Paulo, The Brazilian National Council for Scientific and Technological development, Sarepta Therapeutics, F Hoffmann-La Roche, Biogen, and Novartis. He has received consulting fees from F Hoffmann-La Roche, Novartis, Biogen, Sanofi, Astellas, and Sarepta Therapeutics. EZ received speaker honoraria from and served on advisory boards for F Hoffmann-La Roche, Novartis, Biogen, Sanofi, and Astellas. He has received travel and meeting attendance support from F Hoffmann-La Roche, Novartis, Biogen, Sanofi, and Sarepta Therapeutics. GB reports that he has received consulting fees and speaker honoraria from Biogen, F Hoffmann-La Roche, and Novartis Gene Therapies. GB has received fees for serving on advisory boards and has received equipment for indirect calorimetry to University College London from F Hoffmann-La Roche. CB reports he has received grants or contracts from Biogen, Novartis, and F Hoffmann-La Roche. He has served on advisory boards for Sarepta Therapeutics, F Hoffmann-La Roche, Novartis, and Biogen; he has received support from Sarepta Therapeutics and Biogen for attending meetings. JWD has received research grants from Biogen, Cytokinetics, Ionis Pharmaceuticals, Novartis Gene Therapies, F Hoffmann-La Roche, and Scholar Rock. He reports that he has received consulting fees from Shift Therapeutics and that he has served on advisory boards for Biogen, Cytokinetics, Epirium Bio, Ionis Pharmaceuticals, Novartis Gene Therapies, F Hoffmann-La Roche–Genentech, and Scholar Rock. ND reports he has received support from F Hoffmann-La Roche for provision of FIREFISH and SUNFISH clinical trials study materials, paid to the institution (Ghent University Hospital [US Gent]). He reports that he has received consulting fees for the SUNFISH trial advisory board, has received support for attending meetings, travel, or both, and for serving on advisory boards from F Hoffmann-La Roche. AK has received consulting fees for serving on advisory boards for AveXis, Novartis Gene Therapies, Biogen, and F Hoffmann-La Roche. She has received speaker honoraria from F Hoffmann-La Roche and Biogen. AK reports that The Swiss-Reg-NMD receives unconditional financial support from PTC Therapeutics, Sarepta Therapeutics, Pfizer, and F Hoffmann-La Roche and research grants from Novartis Gene Therapies and Biogen. She reports that she receives research grants from the Swiss Foundation for Research on Muscle Diseases. EM has received grants or contracts from Biogen. He has received speaker honoraria for lectures from Biogen, F Hoffmann-La Roche, Novartis, and AveXis. EM reports he has served on advisory boards for Biogen, F Hoffmann-La Roche, Scholar Rock, Novartis, AveXis, and Cytokinetics. DV reports grants from PTC Therapeutics, F Hoffmann-La Roche, Novartis, Biogen, NS Pharma, Sarepta Therapeutics, and Pfizer. He has received consulting fees from F Hoffmann-La Roche, Novartis, AveXis, and Biogen; he has received speaker honoraria for lectures from PTC Therapeutics, F Hoffmann-La Roche, Janssen, and Novartis. DV has served on advisory boards for AveXis, Biogen, Novartis, and F Hoffmann-La Roche. YW reports she has received payment from F Hoffmann-La Roche for the FIREFISH part 2 trial to support the study according to agreement and has received grants or contracts from UCB and Biogen for clinical trial support. BD reports grants from F Hoffmann-La Roche during the conduct of the study; and grants from PTC Therapeutics, Fibrogen, AveXis, Genentech, Ionis Pharmaceuticals, Biogen, Sarepta Therapeutics, and Summit. He reports research support from the National Institutes of Health and National Institute of Neurological Disorders and Stroke, Slaney Family Fun for spinal muscular atrophy, Spinal Muscular Atrophy Foundation, CureSMA, and Working on Walking Fund. He served on advisory boards for AveXis, Vertex, Genentech, F Hoffmann-La Roche, Sanofi–Genzyme, Sarepta Therapeutics, and Biogen; he reports his role as a member of the data safety monitoring board for Amicus Inc, outside of the submitted work. AD, MEK, KG, BJ, HK, EG, and PF report that they are current employees and stockholders in F Hoffmann-La Roche.

Comment in

New results for risdiplam in spinal muscular atrophy.
Iannaccone ST. Iannaccone ST. Lancet Neurol. 2022 Dec;21(12):1065-1066. doi: 10.1016/S1474-4422(22)00427-6. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244363 No abstract available.

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Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

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Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

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