Drug Discovery of Rare Diseases

Rare disease research has reached a listing point, with the confluence of scientific and technologic developments that if appropriately connected, could lead to key innovations and treatments for this set of devastating conditions. Industry-wide trends have discovered that the traditional drug discovery research and development (R&D) model is no longer viable, and drug companies are developing their approach. Rather than only pursue blockbuster therapeutics for heterogeneous, common diseases, drug companies have gradually begun to shift their focus to rare diseases. In academia, advances in genetics analyses and disease mechanisms have allowed scientific understanding to mature, but the shortage of funding and translational capability severely limits the rare disease research that leads to clinical trials

  • Hepatocyte-targeted RNAi Therapeutics
  • Developing Therapeutics for Major Unmet Medical Needs
  • Challenges of Rare Disease Drug Development
  • Drug for Familial Amyloid Polyneuropathy
  • Rare hematological malignancies
  • New Therapeutic Modalities
  • New Therapeutic Modalities
  • New Therapeutic Modalities
  • New Therapeutic Modalities
  • New Therapeutic Modalities
  • Rare Diseases Clinical Research

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