Preliminary Program

DAY 1

Scientific Program:

  • Basic Science: Disease Mechanisms, Pathology and new Biomarkers of MPS
  • Newborn screening

Family Program:

General discussion: engaging with families, patients, clinicians, geneticists, genetic counsellors on clinical and molecular diagnosis, clinical care persons, experience with individual cases, therapies and clinical biomarkers for assessing outcomes

DAY 2

Scientific Program:

  • New lysosomal disorders and new therapies
  • Animals pre-clinical trial/ humans clinical trial
    • Gen therapy
    • BMT (Bone Marrow Transplant)
    • Stem cells
    • ERT (Enzyme Replacement Therapy)
  • ERT and antibodies I, II, IV, VI, VII
  • Nanoparticles & Other treatments

Family Program:

  • Symptomatic Treatments
    • Growth delay and Growth Hormone
    • Musculoskeletal problems and treatment
    • Cardiac problems and treatment
    • Mental and behavior problems and treatment
    • Anesthetic, what do we have we to take into account/monitor?
    • Best practice in transition from children to adults
    • Epidemiologic studies

DAY 3

  • Late Breaking News on:
    • MPS Research
    • Therapies
    • Treatments
  • General Discussion Q&A
  • Meeting Summary
  • Closing of Meeting