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1385 news items
Research
Life expectancy is increasing for DMD thanks to the angiotensin-converting enzyme (ACE) inhibitors
On 12/04/2021
In Duchenne muscular dystrophy (DMD), heart involvement leads to heart failure. Angiotensin-converting enzyme (ACE) inhibitors are routinely prescribed at around 10 years of age for…
Research
Identification of a slow and gradual decline in muscle strength in GNE myopathy and tailored tools to evaluate it
On 06/04/2021
Characterised by distal muscle weakness, GNE myopathy (also known as Nonaka myopathy, hereditary inclusion body myositis, distal myopathy with rimmed vacuoles or quadriceps-sparing myopathy) is…
Research
Preliminary results of FIREFISH, a trial evaluating ridisplam in type 1 SMA
On 11/05/2020
The primary endpoint of the second part of the FIREFISH clinical trial has been met. This is an announcement by Roche and PTC Therapeutics, the…
Research
Drug-induced myopathies: beware of the toxic effects of immune control point inhibitors
On 03/03/2020
Drug-induced myopathy is among the most common causes of muscle disease. An association has recently been described between programmed death-1 (PD-1)/PD-1 ligand (PD-L1) inhibitors and…
Research
Expanding the phenotype of filamin-C-related myofibrillar myopathy
On 07/05/2019
A tean of Danish researhers reports three patients with a rare filamin C myofibrillar myopathy. They present with atypical symptoms that expand the phenotype of…