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1385 news items
Research
Not all immune checkpoint inhibitor-induced myositis are alike
On 09/05/2023
Spanish and American researchers studied the clinical and serological data of patients with autoimmune myositis in relation to immune checkpoint inhibitor (ICI) treatment. Transcriptomic analysis…
Research
Cardiac involvement is possible in autoimmune necrotizing myopathies
On 05/05/2023
Chinese researchers studied all their cases of autoimmune necrotizing myopathy (AINM) over the period 2011-2022 with a focus on possible cardiac complications: Fifty-seven patients…
Research
Using patient fibroblasts to understand inclusion body myositis
On 05/05/2023
A Spanish team compared the fibroblasts of 14 patients with inclusion body myositis (IBM) to 12 healthy controls. The IBM fibroblasts were found to have:…
Research
Intravenous cyclophosphamide is more effective than other immunosuppressive drugs in interstitial lung disease associated with myositis
On 05/05/2023
A comparison of the efficacy of cyclophosphamide as first-line therapy versus other immunosuppressive therapies (azathioprine, glucocorticoids, mycophenolate mofetil, calcineurin inhibitors, rituximab) was performed in 47…
Research
Two unusual phenotypes reported in inclusion body myositis
On 04/05/2023
Inclusion body myositis (IBM) is an autoimmune myopathy characterised mainly by damage to the flexor digitorum and quadriceps muscles occurring after the age of fifty.…
Research
Phase 2 trial concludes in favour of subcutaneous administration of immunoglobulins in myasthenia gravis
On 04/05/2023
The background treatment of myasthenia gravis (MG) requires, in some cases, the administration of polyvalent immunoglobulins. US clinicians report the results of a multicentre phase…
Research
Genetic analysis strategy in hereditary neuropathies
On 04/05/2023
French clinicians, aiming to guide the genetic diagnosis of patients with hereditary peripheral neuropathy, have outlined in a review the clinical and neurophysiological aspects as…
Research
Publication of results from the IONIS-DMPKRx trial in DM1
On 03/05/2023
IONIS-DMPKRx, also known as baliforsen is an antisense oligonucleotide developed by IONIS Pharmaceuticals in DM1. It was tested in a Phase I/II clinical trial in…
Research
Differentiated complement activation profiles in myasthenia gravis
On 02/05/2023
German and English researchers studied the profile of complement activation in patients with myasthenia gravis, both seropositive and seronegative, and with and without standard immunosuppressive…
Research
A plea for less invasive muscle biopsy techniques
On 02/05/2023
Australian researchers reviewed the medical literature on the practice of muscle biopsy. In particular, their interest and performance at the diagnostic level were studied, as…
Research
Long-term maintenance of normalized cardiac function on ERT in infantile Pompe disease
On 24/04/2023
Analysis of echocardiograms of 27 patients with infantile Pompe disease on enzyme replacement therapy (ERT) obtained during a median follow-up of 9.9 years (maximum 22…
Research
Kennedy disease is at least four times more common
On 21/04/2023
The search for CAG expansion in the AR gene that encodes the androgen receptor using the ExpansionHunter computer tool (a “expansion tracker”) in the genomes…
Research
Treated myasthenia can still cause significant difficulties in everyday life
On 20/04/2023
The international MyRealWorldMG study collects real-life data from adults with autoimmune myasthenia undergoing treatment. Each month, they answer different patient-reported outcome measures (PROM), specific or…
Research
Distal Udd titinopathy: a 15-year natural history
On 17/04/2023
A Finnish retrospective study included data from 137 individuals with distal dominant myopathy, linked to the FINmaj mutation in the TTN gene, followed for 15…
Research
Abatacept has variable success in refractory myositis
On 17/04/2023
The AID trial evaluated the addition of abatacept to standard therapy in 10 participants aged seven to 17 years with refractory juvenile dermatomyositis in an…
Research
NEB gene-related nemaline myopathy: description of a cohort of 33 patients
On 14/04/2023
A cross-sectional study of 33 North American and Brazilian patients with NEB gene-related nemaline myopathy, aged two to 59 years, was published in January 2023.…
Research
Gene therapy confirms its efficacy in the mouse model of limb-girdle muscular dystrophy related to SGCG
On 12/04/2023
A study sponsored by Sarepta Therapeutics investigated the safety and efficacy of SRP-9005 (rAAVrh74 vector), one of their gene therapy products, when injected intravenously into…
Research
Mixed results for givinostat in Becker myopathy
On 11/04/2023
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…
Research
Quality of life of patients dependent on ventilatory support
On 07/04/2023
The AFM-Telethon teams conducted a study of 119 patients with neuromuscular diseases (mainly Duchenne muscular dystrophy but also limb-girdle muscular dystrophy, congenital muscular dystrophy, proximal…
Research
Kennedy’s disease: targeting LSD1 and PRMT6 overexpression attenuates mutated androgen receptor toxicity without worsening androgen deficiency
On 07/04/2023
Lysine-specific demethylase 1 (LSD1) and protein arginine N-methyltransferase 6 (PRMT6) are co-regulators of the androgen receptor. Overexpressed by androgens specifically in skeletal muscle of mice…