Out-of-Frame Mutations in ACTN2 Last Exon Cause a Dominant Distal Myopathy With Facial Weakness.
Marco SavareseAnna ViholaManu E JokelaSanna Pauliina HuovinenSimonetta GereviniAnnalaura TorellaMridul JohariMarina ScarlatoPer Harald JonsonMaria Elena OnorePeter HackmanMathias GautelVincenzo NigroPrevitali Stefano CarloBjarne UddPublished in: Neurology. Genetics (2021)
Our findings indicate an unusual form of actininopathy with specific molecular and clinical features. Actininopathy should be considered in the differential diagnosis of distal myopathy combined with facial weakness.