WMS 2020: an unprecedented edition that took place “online”!

It is in the current, unprecedented health context that the World Muscle Society (WMS) organised its first 100% virtual Congress for the 25th edition. From 28 September to 2 October 2020, researchers, clinicians and other muscle experts were invited to connect to a platform, to listen and then participate in video-conferences, view “e-Posters”, follow symposia organised by pharmaceutical companies, etc.

This edition was an opportunity to review the latest medical and scientific advances in the field of neuromuscular disease, such as:

  • the identification of new genes: the SMPX gene for a first description of X-linked distal myopathy, the JAG2 gene implicated in a new form of limb-girdle muscular dystrophy showing autosomal recessive transmission, etc.,
  • the presentation of new genetic, proteomic and algorithmic approaches to assess neuromuscular disease,
  • a large-scale clinical study suggesting that one should consider LGMD R2 (linked to a mutation of the DYSF gene) and Miyoshi myopathy (also linked to a mutation of the DYSF gene) clinically as a single disease, dysferlinopathy, for future clinical trials,
  • the presentation of fundamental research work or therapeutic advances by Institute of Myology scientists,
  • results of the first gene therapy trial aiming to skip a surplus exon in participants with duplicated exon 2 in the DMD gene, with re-expression of “full-length” dystrophin in small quantities upon muscle biopsy, 3 months after the injection.

Results of gene therapy clinical trials
This virtual Congress also gave rise to discussions about gene therapy, with questions still unresolved, especially regarding immune response and product toxicity, but also encouraging results in Duchenne muscular dystrophy, limb-girdle muscular dystrophy (LGMD), SMN1-related proximal spinal muscular atrophy (SMA), etc.

  • In LGMD R4 (or LGMD 2E): good tolerability of SRP-9003 (which delivers the SGCB gene) and functional improvement 18 months after injection of the 3 participants receiving a low dose, and 6 months after injection of the 3 participants receiving a high dose.
  • In SMA: beneficial effects of Zolgensma® (which delivers the SMN1 gene) on motor development, ongoing, in type 1 SMA (STR1VE-EU trial), including in participants who have symptoms more severe than participants in other studies.
  • In DMD: good tolerability and long-lasting functional improvement for SRP-9001 (microdystrophin) in 4 participants, two years after administration.See you in September 2021 for the 26th edition of the WMS in Prague, fingers crossed!

 

Access World Muscle Society press releases:

 

« Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After Administration »

 

« Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001, its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy »

 

« Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function and milestone achievement »