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Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.

Marisa E SchwabJulia E H BrownBillie LianoglouChengshi JinPatricia C ConroyRenata C GallagherPaul HarmatzTippi C MacKenzie
Published in: Orphanet journal of rare diseases (2022)
Familiarity with postnatal ERT increased respondents' likelihood of pursuing fetal therapies. Families who use clinicaltrials.gov may be more receptive to innovative fetal treatments. The patient community has a favorable attitude towards fetal therapy; over half of respondents would enroll in a phase I clinical trial to assess the safety and efficacy of fetal ERT.
Keyphrases
  • clinical trial
  • end stage renal disease
  • mental health
  • healthcare
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • randomized controlled trial
  • bone marrow
  • mesenchymal stem cells