Works matching IS 22143599 AND DT 2025 AND VI 12 AND IP 2

Results: 12

Long-term safety of cyclical rozanolixizumab in patients with generalized myasthenia gravis: Results from the Phase 3 MycarinG study and an open-label extension.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 231, doi. 10.1177/22143602241308181

By:

Habib, Ali A;
Drużdż, Artur;
Grosskreutz, Julian;
Mantegazza, Renato;
Sacconi, Sabrina;
Utsugisawa, Kimiaki;
Vu, Tuan;
Vissing, John;
Gayfieva, Maryam;
Pulido-Valdeolivas, Irene;
Tarancón, Thaïs;
Woltering, Franz;
Bril, Vera

Publication type:

Article

Rozanolixizumab in generalized myasthenia gravis: Pooled analysis of the Phase 3 MycarinG study and two open-label extensions.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 218, doi. 10.1177/22143602241305511

By:

Bril, Vera;
Drużdż, Artur;
Grosskreutz, Julian;
Habib, Ali A;
Mantegazza, Renato;
Sacconi, Sabrina;
Utsugisawa, Kimiaki;
Vu, Tuan;
Boehnlein, Marion;
Greve, Bernhard;
Gayfieva, Maryam;
Woltering, Franz;
Tarancón, Thaïs;
Vissing, John

Publication type:

Article

A novel variant in VMA21 causing adult-onset phenotype of X-linked myopathy with excessive autophagy with cardiac involvement in a Chinese patient.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 285, doi. 10.1177/22143602241305510

By:

Chen, Lin;
Fang, Ming-juan;
Xu, Dabing;
Shi, Yong-guang;
Han, Yong-zhu;
Yu, Xu-en;
Xu, Yin

Publication type:

Article

A rare case of myopathy with fatigability due to PYROXD1 variation.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 293, doi. 10.1177/22143602241301635

By:

Baskar, Dipti;
Thomas, Aneesha;
Boddu, Vijay Kumar;
Santhoshkumar, Rashmi;
Anjanappa, Ram Murthy;
Nashi, Saraswati;
Srivastava, Kosha;
Polavarapu, Kiran;
Arunachal, Gautham;
Kotambail, Ananthapadmanabha;
Rao, Bhoomika;
Mahadevan, Anita;
Nalini, Atchayaram;
Vengalil, Seena

Publication type:

Article

Systematic literature review of the impact of spinal muscular atrophy therapies on bulbar function.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 195, doi. 10.1177/22143602241303373

By:

McGrattan, Katlyn;
Walsh, Katie;
Mehl, Lesa;
Kaur, Simarjeet;
Dilly, Keith W

Publication type:

Article

Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 173, doi. 10.1177/22143602241303357

By:

Kaufman, Beth D.;
Veerapandiyan, Aravindhan;
Soslow, Jonathan H;
Wittlieb-Weber, Carol;
Esteso, Paul;
Olson, Aaron K.;
Shih, Renata;
Bansal, Neha;
Lal, Ashwin;
Gambetta, Katheryn;
Hsu, Daphne;
Cripe, Linda;
Villa, Chet;
Nandi, Deipanjan

Publication type:

Article

A rare homozygous CAPN3 variant with distinct clinical features in unrelated families of Iraqi Jewish descent.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 279, doi. 10.1177/22143602241301658

By:

Assia Batzir, Nurit;
Orenstein, Naama;
Yaron, Yuval;
Kuzminsky, Alla;
Nevo, Yoram;
Konen, Osnat;
Bazak, Lily;
Lidzbarsky, Gabriel;
Basel-Salmon, Lina;
Aharoni, Sharon

Publication type:

Article

Analysis of spinal muscular atrophy patients from the spinal muscular atrophy and muscular dystrophy registry of Pakistan.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 260, doi. 10.1177/22143602241301657

By:

Aziz, Bisma;
Arif, Ahmed A;
Kazi, Kulsum;
Kirmani, Salman;
Ansar, Zeeshan;
Nasir, Asghar;
Ibrahim, Shahnaz Hamid;
Ahmed, Khairunnisa Mukhtiar;
Hasan, Zahra;
Khan, Sara

Publication type:

Article

Plasma-derived protein and imaging biomarkers distinguish disease severity in oculopharyngeal muscular dystrophy.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 244, doi. 10.1177/22143602241304990

By:

Smith, Ian C;
Sampaio, Marcos L;
Melkus, Gerd;
Meier-Ross, Kaitlynn;
Chakraborty, Shaoni;
Stotts, Cameron;
Bourque, Pierre R;
Lochmuller, Hanns;
Brais, Bernard;
Ayoub, Othmane;
Perkins, Theodore J;
Khacho, Mireille;
Warman-Chardon, Jodi

Publication type:

Article

Newborn screening and rapid genomic diagnosis of neuromuscular diseases.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 157, doi. 10.1177/22143602241296286

By:

Dangouloff, Tamara;
Lang, Helena;
Benmhammed, Noor;
Servais, Laurent

Publication type:

Article

Glycogenosis type XI, a rare association between muscle and skin manifestations - the contribution of proteomics for the understanding of the underlying myopathology.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 271, doi. 10.1177/22143602241296248

By:

Hentschel, Andreas;
Lacene, Emmanuelle;
Brochier, Guy;
Boisserie, Jean-Marc;
Fromes, Yves;
Romero, Norma Beatriz;
Roos, Andreas;
Evangelista, Teresinha

Publication type:

Article

Ultra-Orphan drug development for GNE Myopathy: A synthetic literature review and meta-analysis.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 2, p. 183, doi. 10.1177/22143602241296226

By:

Suzuki, Naoki;
Mori-Yoshimura, Madoka;
Nishino, Ichizo;
Aoki, Masashi

Publication type:

Article