Clinical trial readiness study of distal myopathy and dysphagia in nephropathic cystinosis.
Reza SadjadiStacey SullivanNatalie GrantSusan E ThomasMaya DoyleColleen HammondCamille CorreNicholas MelloWilliam S DavidFlorian EichlerPublished in: Muscle & nerve (2020)
Improved respiratory outcomes may enhance patients ability to expel aspirated material from the airway, stave off pulmonary sequelae associated with chronic aspiration, and yield an overall improvement in physical health and well-being.
Keyphrases
- clinical trial
- end stage renal disease
- mental health
- newly diagnosed
- public health
- chronic kidney disease
- physical activity
- pulmonary hypertension
- prognostic factors
- peritoneal dialysis
- randomized controlled trial
- late onset
- minimally invasive
- ultrasound guided
- open label
- adipose tissue
- study protocol
- phase ii
- social media
- double blind
- early onset
- patient reported