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In line with their research over several decades and the advances they have already spearheaded, Professors Olivier Benveniste and Yves Allenbach (the Inflammatory Myopathies & Innovative Targeted Therapies team at the Center for Research in Myology of the Institute of Myology)…
In 13 countries, including France, the Phase III Nimble trial evaluated cemdisiran, a small interfering RNA (siRNA) targeting complement component 5, administered subcutaneously, in patients with generalised myasthenia gravis with anti-RACh and/or anti-LRP4 antibodies: 263 adults were treated for 24…
One therapeutic approach for Duchenne muscular dystrophy (DMD) involves increasing the expression of utrophin to compensate for the absence of dystrophin, with which it shares a high degree of homology. A team at Généthon used the CRISPR-Cas9 system to generate…
The team at Great Ormond Street Hospital in London compiled clinical and molecular data from patients suspected of having congenital myopathy or congenital muscular dystrophy (CMD): all had undergone high-throughput sequencing (NGS) genetic testing; of the 1,927 individuals studied with…
Episode 4 – Infectious and Neoplastic Neuropathies (in EURO-NMD Neuropathology Webinar Series) Thursday 7 May 2026 – 16:00-17:00 Paris time Speaker: Prof. Mathilde Duchesne (CHU Limoges, France) Moderators: Dr. Matteo Garibaldi (Sant’Andrea Hospital of Sapienza University of Rome, Italy), Prof. Joachim Weis(Director,…
An international consortium of experts on facioscapulohumeral muscular dystrophy (FSHD) has compiled observations from patients diagnosed at a relatively advanced age: using clinical and biological data from a previous clinical trial called RESOLVE, the group identified 41 patients with late-onset…
US experts in gene therapy for Duchenne muscular dystrophy (DMD) have investigated the causes of two deaths that occurred in 2025 and were attributed to the administration of a single dose of dilandistrogene moxeparvovec (Elevidys®). They propose the following preventive…
A tean of Danish researhers reports three patients with a rare filamin C myofibrillar myopathy. They present with atypical symptoms that expand the phenotype of filaminopathy. The new findings are progressive contractures of muscles surrounding the temporomandibular joint, detailed single…