Blog Archives
Casimersen examined in a phase I/II trial in limited ambulatory or non-ambulatory DMD patients is well tolerated and eliminated over time
Casimersen (SRP-4045, Amondys45), an antisense oligonucleotide targeting exon 45 skipping of the DMD gene, is one of the exon skipping agents developed in Duchenne muscular dystrophy (DMD) by Sarepta Therapeutics. It obtained conditional marketing authorisation (MA) in the United States in February 2021. A phase I/II US trial studied this agent in limited ambulatory or non-ambulatory … [Read more]
DMC related to LMNA: on a large-scale retrospective natural history
A retrospective international study coordinated by a team from the Myology Centre for Research at the Institute of Myology was conducted to describe the natural history of an early-onset form of laminopathy: congenital muscular dystrophy linked to the LMNA gene. Thus, data from 151 patients with laminopathy who presented with muscle symptoms that appeared before … [Read more]
The last congress of the FSHD Society took place on June 24 and 25, 2021
Gathered online, facio-scapulo-humeral myopathy (FSH) experts from around the world shared and updated on the latest medical-scientific advances, both in the field of research and clinical trials. The various presentations provided an opportunity to discuss in particular: the development of a new FSH mouse model: it can express very low levels of DUX4 under … [Read more]
Decrease the time from diagnosis to the start of the treatment with Treatabolome
The creation of an open platform drawing up an inventory of all existing treatments for rare genetic diseases, including neuromuscular diseases, is one of the objectives of the European Solve-RD project, which fights diagnostic delay. This platform, better known as the “Treatabolome”, should give clinicians better visibility and knowledge of the treatments available depending on … [Read more]
Release of the 86th newsletter from the Institute – July 2021
Prof. Fabrice Chrétien joined the teams of the Institute of Myology on June 15 as Director of the Center for Neuromuscular Exploration and Assessment which is currently composed by a staff of thirty persons. It encompasses three laboratories and a tissue bank: the Neuromuscular Physiology and Evaluation Laboratory, the NMR and Spectroscopy Laboratory, the Morphological Unit and the biological … [Read more]
Appointment of Piera Smeriglio at the head of BOND group
Piera Smeriglio will take over the management of the BOND Group – Biotherapies for motor neuron disorders (ALS & SMA) following the upcoming departure of Maria Grazia Biferi to whom we send our best wishes and whom we warmly thank for the work she has done with her group within the framework of the Myology … [Read more]
DMD: start at I-Motion of trials with pamrevlumab in ambulatory and non-ambulatory patients
I-Motion is recruiting patients with Duchenne muscular dystrophy to participate in two pharmacological trials designed to test an antifibrotic monoclonal antibody, pamrevlumab (or FG-3019), developed by FibroGen. The objective of these phase III trials is to assess the tolerance, safety and efficacy on the upper limbs, respiratory and cardiac functions of pamrevlumab administered by intravenous … [Read more]
Success for AcadeMYO, the first virtual edition of the Myology Summer School
AcadeMYO, the first fully virtual release of the institute’s Myology Summer School, took place July 5-9. This 2021 edition was a great success. In fact, 115 people attended the courses – nearly twice as many students as during the last face-to-face edition in Paris – including 15 free auditors and 6 representatives of the pharmaceutical … [Read more]
Prof. Fabrice Chrétien joins the Institute of Myology to head the Neuromuscular Exploration and Evaluation Centre
With mixed medical and scientific training, Prof. Fabrice Chrétien has more than 20 years of medical experience in anatomopathology and more than 30 years of research experience in neuropathology. Fabrice Chrétien joined the teams of the Institute of Myology on June 15 as Director of the Neuromuscular Investigation Center currently having around thirty people. It encompasses … [Read more]
Muscle imaging is useful in the diagnosis and follow-up of most autoimmune myositis
Idiopathic inflammatory myopathies constitute a heterogeneous group of muscular diseases composed, basically, of four major groups: dermatomyositis, overlapping myositis (including anti-synthetase syndrome), inclusion myositis (IBM) and self-necrotizing myopathies. immune. Their diagnosis is based, according to recent international recommendations, on muscle biopsy data and specific auto-antibody assays (MSA for myositis specific auto-antibodies). In an article published … [Read more]
