Open‐label, single‐center, clinical study evaluating the safety, tolerability and clinical effects of pentosan polysulfate sodium in subjects with mucopolysaccharidosis I

Documenti analoghi

• Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase‐ IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II
  di: Joseph Muenzer, et al.
  Pubblicazione: (2024)
• Natural history of valve disease in patients with mucopolysaccharidosis II and the impact of enzyme replacement therapy
  di: Christoph Kampmann, et al.
  Pubblicazione: (2024)
• Development of a novel tool for individual treatment trials in mucopolysaccharidosis
  di: Anna‐Maria Wiesinger, et al.
  Pubblicazione: (2024)
• Long‐term cardiovascular outcomes and mortality with enzyme replacement therapy in patients with mucopolysaccharidosis type II
  di: Ji Hee Kwak, et al.
  Pubblicazione: (2024)
• Clinical pharmacology considerations for first‐in‐human clinical trials for enzyme replacement therapy
  di: Sydney Stern, et al.
  Pubblicazione: (2024)