Muscle and Bone
The effects of palovarotene in patients with fibrodysplasia ossificans progressiva: a plain language summary
This plain language summary from Future Rare Diseases describes the results of the MOVE study, which investigated the drug palovarotene in the treatment of patients with fibrodysplasia ossificans progressiva (FOP).
The impact of fibrodysplasia ossificans progressiva (FOP) on patients and their family members: results from an international burden of illness survey
Learn about the results of the first international fibrodysplasia ossificans progressiva burden of illness survey in this plain language summary.
Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study
This plain language summary of publication, co-authored by a patient, a caregiver and a treating physician, includes the patient perspective and help patients with Duchenne muscular dystrophy and their caregivers understand the results of the Cincinnati study.
Plain language summary of the long-term effect of ataluren in patients with Duchenne muscular dystrophy
Read the latest plain language summary looking at how ataluren treatment in combination with standard of care can delay important signs of disease progression in patients with Duchenne muscular dystrophy.
Vosoritide treatment accelerates bone growth in children with achondroplasia
Read the article summarising the results of two studies looking at vosoritide as a potential treatment for children with achondroplasia.