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1385 news items
Research
Compliance with non-invasive ventilation is not related to respiratory parameters in DM1
On 15/12/2023
Dutch researchers studied compliance with non-invasive mechanical ventilation (NIV) prescribed as part of respiratory management for patients with Steinert’s disease (DM1). 101 patients who had…
Research
Low-carb ketogenic diet and McArdle’s disease: results of an international survey
On 15/12/2023
An international survey was conducted among 183 people with McArdle’s disease in 18 countries, including France, to gather their experiences with the low-carbohydrate, high-fat ketogenic…
Research
Duchenne muscular dystrophy: the possibility of necroptosis inhibition
On 14/12/2023
In the dystrophin-deficient muscle of mouse, rat and dog models of Duchenne muscular dystrophy, there is activation of necroptosis, RIPK1 and RIPK3, key signaling molecules…
Research
A CRISPR-Cas9 system induces multiple exon skipping in DMD
On 13/12/2023
Genome editing is an emerging approach to the targeted treatment of Duchenne muscular dystrophy (DMD). Japanese researchers have conducted experiments using the CRISPR-Cas9 tool in…
Research
Myopathology of DMD reveals intrinsic senescence of muscle stem cells
On 13/12/2023
A Franco-Italian team has studied the regeneration processes, fibro-adipogenic precursors and muscle stem cells in 24 muscle biopsies from patients with Duchenne muscular dystrophy (DMD):…
Research
Corticosteroids improve the efficacy of preventive treatment of left ventricular failure in DMD
On 12/12/2023
A retrospective study of the occurrence of left ventricular damage in 455 boys with Duchenne muscular dystrophy, born between 1982 and 2009 and followed for…
Research
A large-scale natural history study of Becker’s myopathy
On 11/12/2023
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…
Research
Growth hormone to offset corticosteroid-induced growth retardation in DMD
On 10/12/2023
Four boys suffering from Duchenne muscular dystrophy treated with corticosteroids (deflazacort or prednisone) had growth retardation with height varying between -3.24 and -1.85 standard deviations,…
Research
Identification of the causes of death of the only patient treated in the first CRISPR-Cas9 gene therapy trial for DMD
On 10/12/2023
American researchers have analysed the causes of death of a 27-year-old Duchenne muscular dystrophy (DMD) patient who took part in the first trial using the…
Research
When a smartphone app reliably assesses peak cough flow
On 08/12/2023
A study carried out in Spain on 50 patients suffering from Charcot’s disease (n=26), autoimmune myasthenia (n=9) or genetic myopathy (n=15) shows that sound analysis…
Research
Treatment of thymoma with immune checkpoint inhibitors increases the risk of muscle toxicity
On 07/12/2023
An analysis of various registries and clinical trial data, carried out by an international team involving researchers from the Institute of Myology, revealed: 1,495 cases…
Research
Pre-injection aerobic exercise increases the effectiveness of gene therapy
On 07/12/2023
American researchers have investigated the benefits of a short, low-intensity physical activity session on the efficacy of gene therapy product transduction in mouse models of…
Research
Scoliosis associated with early muscle weakness should prompt a search for CMS
On 06/12/2023
This is the conclusion of an article by a Turkish team which reviewed the records of eleven patients with congenital myasthenic syndrome presenting with scoliosis:…
Research
GNE myopathy and 6′-sialyllactose: mixed results
On 06/12/2023
A South Korean team conducted a randomised trial of 6′-sialyllactose (6SL) versus placebo in GNE myopathy for almost two years (96 weeks) in 20 participants…
Research
Painful muscle cramps at the forefront in a very rare form of hereditary sensory-motor neuropathy
On 05/12/2023
Okinawa-type neurogenic muscular atrophy (or HMSN-P) is an extremely rare hereditary neuropathy. Japanese researchers have taken a new interest in what is one of the…
Research
DMD: first human study of DT-DEC01 cell therapy
On 04/12/2023
A Polish-American team working in collaboration with the laboratory Dystrogen Therapeutics has published the results of administering a new cell therapy product, DT-DEC01, to patients…
Research
DREAMS – a new Horizon Europe project brings hope to rare disease patients
On 29/11/2023
The objective of this 5-year project is to discover treatments for 5 rare neuromuscular disorders through a groundbreaking methodology combining Artificial intelligence (AI), stem cells…
Research
Garetosmab is effective in fibrodysplasia ossificans progressiva
On 27/11/2023
The Phase II LUMINA 1 trial assessed the safety and efficacy of garetosmab, a monoclonal antibody that blocks activin A signaling pathways, in 44 adults…
Research
Facial involvement is common in inclusion myositis
On 24/11/2023
Following on from previous work on facioscapulohumeral muscular dystrophy (FSHD), researchers in Marseilles have studied the characteristics of facial muscle deficit in people with inclusion…
Research
Testosterone in DMD improves the situation but does not normalise it
On 23/11/2023
The Newcastle team looked at the long-term follow-up of a cohort of 15 boys with Duchenne muscular dystrophy on corticosteroids who were treated with increasing…