International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Drug Discovery and Developments

Drug Discovery and Developments

Pharmaceuticals used for the diagnosis, prevention, and treatment of rare diseases are known as orphan drugs. Rare diseases differ from common diseases in that they have a low prevalence rate and so appeal to a small patient population. These medications are being developed to treat people with extremely rare disorders for which there is now no therapy option. Pharmaceutical inventors are often discouraged from developing treatments for extremely limited patient groups due to the increasing expense of medication research in tandem with rigorous regulations, as well as the low return on investment. Orphan diseases are rare diseases that affect small patient groups and are consequently "orphaned" by the pharmaceutical industry, with only a few authorized pharmacological therapy choices available. Incentives for orphan drugs have sparked research into diseases with a high unmet medical need.

  • Clinical Insight
  • Drug Clinical Status
  • Global Market Opportunity
  • Orphan Clinical Pipeline
  • Drug Class & Formulation
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Vicki Ratner

Vicki Ratner

ESSIC - International Society for the Study of BPS, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Kondakova O. B

Kondakova O. B

National Medical Research Center for Children's Health Federal state autonomous institution of the Russian Federation Ministry of Health, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2023 - Paul L Kaufman

Paul L Kaufman

University of Wisconsin-Madison, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Magali Taiel

Magali Taiel

GenSight Biologics, France

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