Becker muscular dystrophy
RSS feedDMD: Commercial go-ahead for givinostat (Duvyzat™) in the USA
Givinostat, a drug developed by Italfarmaco for Duchenne and Becker muscular dystrophies, is a histone deacetylase (HDAC) inhibitor. On 21 March 2024, it was granted marketing authorisation in the United States by the FDA on the basis of the results of the EPIDYS phase III double-blind, placebo-controlled trial, which took place in France. The EPIDYS … [Read more]
Sleep disordered breathing in slowly progressive myopathies: beyond alveolar hypoventilation
A French retrospective study of the records of 149 adults with an average age of 46.5 years and suffering from myotonic dystrophy (45) , myasthenia (20), Pompe disease (9), spinal muscular atrophy (8), facioscapulohumeral myopathy (8), Duchenne myopathy (5), Becker myopathy (1) or another neuromuscular disease (53) explored by respiratory polygraphy, nocturnal transcutaneous capnography and … [Read more]
A large-scale natural history study of Becker’s myopathy
The records of 225 Japanese patients with Becker’s myopathy, with an average age of 31, were examined to gain a better understanding of the natural history of this disease. The results show that : muscular dystrophy begins with muscular symptoms, hyperCKemia and central nervous system disorders, 53.8% of patients have walking difficulties, with wheelchair use … [Read more]
Mixed results for givinostat in Becker myopathy
A phase II, double-blind, placebo-controlled clinical trial evaluated the efficacy and safety of givinostat in 51 adults with Becker muscular dystrophy (BMD). According to the results after one year of treatment, published in January 2023 : givinostat had no effect on total muscle fibrosis, the primary endpoint of this study. magnetic resonance imaging (MRI) showed … [Read more]
How American families affected by Duchenne or Becker muscular dystrophy feel about newborn screening
In order to better understand the preferences of families regarding newborn screening, a questionnaire was completed by 66 families affected by Duchenne or Becker muscular dystrophy. The results showed that : the average age at diagnosis was four years, with the onset of symptoms at the age of 2.1 years, the first worrying manifestations were … [Read more]
An ancillary study of the ginivostat trial in Becker myopathy looking for more objective criteria
The primary endpoint for evaluating ginivostat in Becker muscular dystrophy is the change in fibrosis on muscle biopsy after one year of treatment. Based on the data collected at the initial visit of this trial, an Italian and Dutch team looked for correlations between the measurement of the fat fraction of the whole thigh and … [Read more]
