Newsletter #72

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	Newsletter #72

Welcome to our 72nd newsletter! The international congress Myology 2019 officially opens next Monday at Bordeaux and will attend more than 800 experts coming from 35 countries. Each day, you will find on the website of the institute a report of sessions of the day before and interviews with speakers. Until then, please find the scientific programme here. Continuing in International, AFM-Téléthon received the « Pioneer in Technology Development » award for its role in the development of innovative therapies for neuromuscular and rare diseases at the Annual World Congress of Society for Brain Mapping and Therapeutics (SBMT-Brain and Spine). More
At the Institute
The STR1VE EU Trial, a new gene therapy trial in SMA associated with SMN1, is launched in a single center in France, at I-Motion STR1VE EU is an open-label, phase III study to evaluate the efficacy and safety of utilisation of a single, intravenous injection of AVXS-101, in 30 infants with SMA type 1, under 6 months of age. Read more
The paralympic champion Marie-Amelie Le Fur visits the institute The Myology Institute welcomes Marie-Amelie Le Fur, president of the French Paralympics and Sports Committee for Paris 2024. An « inspiring » visit for the athlete. Read more
Our latest news
Nuclear defects in skeletal muscle from a Dynamin 2-linked centronuclear myopathy mouse model This study highlights reduction of number of myonuclei, a key regulator of the myofiber size, as a new pathomechanism underlying muscle atrophy in the dominant centronuclear myopathy. Read more
Diagnosis of respiratory diseases: Development of a new non-invasive method for the evaluation of diaphragm function This new method could represent an alternative to standard invasive methods, which rely on the use of probes inserted into patients’ esophagus and stomach. Read more
Pluripotent stem cell-based drug screening reveals cardiac glycosides as modulators of DM1 These results further underscore the value of stem-cell-based assays for drug discovery in monogenic diseases. Read more
French expert opinion for the management of juvenile dermatomyositis A guideline group elaborated guidelines related to the management of juvenile dermatomyositis on behalf of the rare autoimmune and autoinflammatory diseases network FAI2R. Read more
More breaking news
Preliminary results for microdystrophin SGT-001 gene therapy trial in DMD A modest production of dystrophin from 3 muscular biopsies in three participates in a phase I/II trial, IGNITE-DMD, after 3 months of treatment with SGT-001. Read more
EGFR-Aurka signaling rescues polarity and regeneration defects in dystrophin-deficient muscle stem cells by increasing asymmetric divisions Through a small-molecule screen, a team of Canadian researchers identified epidermal growth factor receptor (EGFR) and Aurora kinase A (Aurka) as regulators of asymmetric satellite cell divisions. Read more
Duchenne muscular dystrophy Positive results of an Omega-3 trial in DMD A phase I study of TAS-205 in patients with DMD Levosimendan as rescue therapy for acute heart failure in a patient with DMD
Spinal muscular atrophy Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotype Assessment of fatigability in patients with SMA: development and content validity of a set of endurance tests
Inflammatory Myopathies Exercise in Myositis Corneal involvement of patients with polymyositis and dermatomyositis
Other neuromuscular diseases and basic research Genetic landscape and novel disease mechanisms from a large LGMD cohort of 4656 patients Therapeutic regimen of L-arginine for MELAS: 9-year, prospective, multicenter, clinical research
In brief
SMA Europe - Call for SMA Research Projects 2019 This new Call for SMA Projects will be open to any research project aimed at finding a therapy for Spinal Muscular Atrophy (SMA) or at elucidating the basic pathophysiological processes of the disease. Read more
Thematic day on viral vectors - May 17, 2019 Registration are now open for this thematic day wich will take place on May 17, 2019, at ICM on La Pitié Salpêtrière site in Paris. Abstract submission and early bird registration by March 31. Read more
EUSTM-2019 - October 18-20, 2019 The 6th Annual European Congress on Clinical & Translational Sciences (EUSTM-2019), 18-20 October, 2019 in Vienna, Austria. Read more
Paris Summer School of Myology - June 17-22, 2019 The 22nd Summer School of Myology will be held at the Institut de Myologie from Monday June 17 to Saturday June 22, 2019. The deadline for applications is set to March 31, 2019. Read more
Job opportunities The institute of Myology is seeking a Project Manager and a Project Assistant. The University of Sherbrooke (Canada) is offering a Postdoctoral Position in Muscle stem cell biology and regenerative medicine. Read more

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Legal This is the newsletter of current medical science from the Institute of Myology. It is published every two months. Chief Editor: Marie-Pierre Verrier Editorial Board: Marianne Perreau-Saussine; J. Andoni Urtizberea; Bertrand Fontaine. Redaction: Anne Berthomier; Dawn C. Haughton. Also participate: Hala Alameddine. Do you have any questions? Would you like to share some news? Please contact us. © 2019 - AFM - Institut de Myologie. ISSN 1772-9866