Pediatric Disorder Clinical Trial
Official title:
Facioscapulohumeral Dystrophy in Children: a Prospective, Observational Study on the Natural History, Predictors and Clinical Impact (iFocus)
This study will focus on the symptoms, natural history and clinical impact of
facioscapulohumeral muscular dystrophy (FSHD) in children.
Symptoms of classical FSHD start in adulthood. However, a small subgroup of FSHD patients
have an early, childhood onset. This early onset is associated with faster progression and
other symptoms like hearing loss and epilepsy.
The symptoms, natural history and clinical impact of FSHD in children are largely unknown.
The results of this study will be vital for adequate symptomatic management and
trial-readiness.
FSHD is a hereditary muscle disease with slowly progressive muscle weakness. In children it
is a very heterogenic disease ranging from severely affected infants to mildly affected
adolescents. Symptoms can include muscle weakness, pain, fatigue, epilepsy, hearing loss,
vision loss, mental retardation and spinal deformities. The prevalence of these symptoms and
the adequate follow-up of these symptoms is unknown. Moreover the clinical impact and social
functioning of children with FSHD is under exposed.
Therefore this study will focus on the total spectrum of FSHD in children.
In addition, an extensive genetic screening will be conducted, searching for (epi)genetic
disease modifiers and severity predictors.
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