Clinical and imaging spectrum of non-congenital dominant ACTN2 myopathy

Source
Journal of neurology - ISSN 0340-5354-272:2 (2025) p. 1-12
Author(s)
    Pablo Iruzubieta, Jose Verdu-Diaz, Ana Topf, Leonela Luce, Kristl G. Claeys, Willem De Ridder, Lidia Gonzalez-Quereda, Carlos Pablo de Fuenmayor-fernandez de la Hoz, Juan Jose Poza, Miren Zulaica, Peter De Jonghe, Jennifer Duff, Magdalena Mroczek, Paloma Martin-Jimenez, Aurelio Hernandez-Lain, Cristina Dominguez-Gonzalez, Jonathan Baets, Pia Gallano, Jordi Diaz-Manera, Volker Straub, Adolfo Lopez de Munain, Roberto Fernandez-Torron

Heterozygous loss-of-function variants in SPTAN1 cause a novel early childhood onset distal myopathy with chronic neurogenic features.

Source
medRxiv : the preprint server for health sciences- (2024) p.
Author(s)
    Jonathan De Winter, Liedewei Van de Vondel, Biljana Ermanoska, Alice Monticelli, Arnaud Isapof, Enzo Cohen, Tanya Stojkovic, Peter Hackman, Mridul Johari, Johanna Palmio, Megan A Waldrop, Alayne P Meyer, Stefan Nicolau, Kevin M Flanigan, Ana Töpf, Jordi Diaz-Manera, Volker Straub, Cheryl Longman, Catherine A. McWilliam, Rotem Orbach, Sumit Verma, Regina Laine, Sandra Donkervoort, Carsten G. Bonnemann, Adriana Rebelo, Stephan Züchner, Tiffany Grider, Michael E. Shy, Isabelle Maystadt, Florence Demurger, Anita Cairns, Sarah Beecroft, Chiara Folland, Willem De Ridder, Gina Ravenscroft, Gisèle Bonne, Bjarne Udd, Jonathan Baets

A study concept of expeditious clinical enrollment for genetic modifier studies in Charcot-Marie-Tooth neuropathy 1A

Source
Journal of the peripheral nervous system - ISSN 1085-9489-29:2 (2024) p. 202-212
Author(s)
    Isaac R.L. Xu, Matt C. Danzi, Ariel Ruiz, Jacquelyn Raposo, Yeisha Arcia De Jesus, Mary M. Reilly, Andrea Cortese, Michael E. Shy, Steven S. Scherer, David N. Herrmann, Vera Fridman, Jonathan Baets, Mario Saporta, Reza Seyedsadjadi, Tanya Stojkovic, Kristl G. Claeys, Pooja Patel, Shawna Feely, Adriana P. Rebelo, Maike F. Dohrn, Stephan Zuchner

Vitamin D₃ deficiency and osteopenia in spastic paraplegia type 5 indicate impaired bone homeostasis

Source
Scientific reports - ISSN 2045-2322-14:1 (2024) p. 1-9
Author(s)
    Sabrina Ehnert, Stefan Hauser, Holger Hengel, Philip Hoeflinger, Rebecca Schuele, Tobias Lindig, Jonathan Baets, Tine Deconinck, Peter De Jonghe, Tina Histing, Andreas K. Nuessler, Ludger Schoels, Tim W. Rattay