Gene And Cell Therapy For Rare & Common Diseases

There area unit about 7,000 known rare diseases, however solely a number of hundred have treatments square measure approved. Factor medical aid is especially relevant to rare illness patients, as over eighty p. c of rare diseases have a noted heritable (single-gene) cause. Ancient little molecule medicine usually works by minimizing symptoms instead of action the illness. Once treating a chronic condition, this could mean frequent administration of the drug or medicine needed to manage the condition. In distinction, factor medical aid has the potential to correct underlying genetic defects, providing a cure instead of merely managing symptoms. Moreover, triple-crown factor medical aid could need solely one dose to confer womb-to-tomb improvement instead of requiring a time period of in progress treatment.


  • Neurological Disorder
  • Blindness Vision Impairment
  • Orthopedic Repair
  • Cancer
  • Wound Healing
  • HIV
  • Cardiovascular Disorder

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Gene And Cell Therapy For Rare & Common Diseases Conference Speakers