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1385 news items
Research
Vamorolone (Agamree®) authorised in the USA for DMD from the age of 2; a decision expected in Europe soon
On 10/11/2023
Vamorolone is a “dissociative” synthetic steroid developed by Santhera and ReveraGen for Duchenne muscular dystrophy, as an anti-inflammatory treatment alternative to conventional corticosteroids. The FDA…
Research
Metformin may have a protective effect on statin muscle toxicity
On 10/11/2023
This is the finding of a one-year Korean study which compared 4,092 patients on statins + metformin with 8,161 patients on statins alone. Analysis of…
Research
US recommendations on the use of immunoglobulins in MNM
On 09/11/2023
Immunoglobulins are still widely prescribed for certain neuromuscular diseases of autoimmune origin, despite the absence of irrefutable scientific evidence. The scarcity of this type of…
Research
French study highlights neurotoxicity of immune checkpoint inhibitors
On 09/11/2023
Clinicians and researchers belonging to two oncology networks, including one devoted to paraneoplastic syndromes, have studied neurological complications arising during the use of a new…
Research
Institute researchers at the 4th International Imaging in Neuromuscular Disease Conference
On 08/11/2023
The 4th International Imaging in Neuromuscular Disease Conference organized by the MYO-MRI consortium was held in Berlin, Germany, from November 5 to 7, 2023. This…
Research
A very high risk of cardiac complications in Emery-Dreifuss muscular dystrophy
On 08/11/2023
An international consortium of researchers and clinicians investigated disorders of cardiac function and/or cardiac rhythm during the course of Emery-Dreifuss muscular dystrophy linked to the…
Research
Neuromuscular diseases in developing countries: increasing genetic data from under-represented populations
On 07/11/2023
While most (86%) published genetic data on neuromuscular diseases comes from populations of European ancestry, the majority of affected families live in low- to middle-income…
Research
Congenital myasthenic syndromes are common in India
On 07/11/2023
The team of clinicians at Bangalore University Hospital in southern India compiled clinical and genetic data on all cases of congenital myasthenic syndrome (CMS) diagnosed…
Research
A founder effect identified in South Indian patients with beta-sarcoglycanopathy
On 06/11/2023
Beta sarcoglycanopathy is one of the four forms of limb-girdle myopathy associated with sarcoglycan deficiency. Indian clinicians have just identified an outbreak of this disease…
Research
A comparative study of the extra-muscular manifestations of anti-synthetase syndrome and dermatomyositis
On 03/11/2023
As part of the MYONET patient registry, a consortium of clinicians compared the clinical and biological data of adult patients with either antisynthetase syndrome (ASys)…
Research
Clear mitochondrial abnormalities and dermatomyositis: a case report
On 02/11/2023
A team of Italian clinicians report the observation of a 77-year-old woman presenting with a classic picture of dermatomyositis (DM) with positive anti-Mi-2 autoantibodies: study…
Research
Russian success with tofacitinib in FOP
On 02/11/2023
Treatment with tofacitinib 5 mg twice daily was evaluated in 13 children with chronic fibrodysplasia ossificans aged 2.2 to 19.6 years: it reduced the mean…
Research
The French Cannemuss study confirms the efficacy of anti-Covid-19 messenger RNA vaccines in cases of amyotrophy
On 31/10/2023
As the Covid-19 booster vaccination campaign gets underway in France, a new publication of the results of the Cannemuss observational study (supported by the AFM-Téléthon…
Research
FSHD: searching for the ideal biomarker
On 31/10/2023
British and Dutch researchers have joined forces to discover a possible biological marker for facioscapulohumeral muscular dystrophy (FSHD): in view of the disappointing and/or contradictory…
Research
Poor quality sleep in CMT?
On 31/10/2023
The Italian Charcot-Marie-Tooth registry conducted a study of sleep and sleepiness among more than 250 patients: 56% had poor quality sleep. 23% doze off during…
Research
First clinical results of a gene therapy for the treatment of FKRP-related LGMD
On 30/10/2023
The initial results of the ATA-100 gene therapy clinical trial for the treatment of limb-girdle muscular dystrophy linked to the FKRP gene (LGMD2I/R9) were presented…
Research
Camptocormia and a dropped head are poor prognostic factors in certain forms of myositis
On 30/10/2023
An international consortium of clinicians coordinated by the Strasbourg Reference Centre looked at patients with inflammatory myopathy with regard to two uncommon clinical signs: camptocormia…
Research
Challenges facing people with primary mitochondrial myopathy
On 30/10/2023
An article on primary mitochondrial myopathies has been published by the Mitochondrial Medicines Society (MMS) and the United Mitochondrial Disease Foundation (UMDF): it highlights and…
Research
The phenotype of patients with SMA carrying 4 copies of the SMN2 gene varies significantly
On 27/10/2023
The Italian ITASMAC network dedicated to spinal muscular atrophy (SMA) studied 169 individuals, children or adults, with a homozygous deletion of the SMN1 gene and…
Research
A bone marrow tumour discovered late after treatment with Zolgensma®: case report
On 26/10/2023
American clinicians report the observation, unique to date, of a child diagnosed with SMA in the neonatal period and then treated with gene therapy (Zolgensma®…