International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Clinical Research and Developments

Clinical Research and Developments

A rare disease, often known as an orphan disease, is a medical disease that only affects a small percentage of the population. A pharmacological agent developed specifically to treat an orphan disease is known as an orphan drug. The journey from the discovery of a new drug to its commercialization is lengthy (10 years on average), costly (tens of millions of euros), and fraught with risk (among ten molecules tested, only one may have a therapeutic effect). The development of a drug to treat a rare condition does not allow for the recovery of the money spent on research. Over the previous two decades, the percentage of new medicines approved for orphan drugs has risen considerably. Approximately 500 orphan designated medications have been approved for the treatment of a variety of oncological, metabolic, hematologic, immunologic, infectious, and neurological disorders. Patients with rare diseases must be kept up to date on scientific and treatment developments.

  • Clinical Trial Transparency
  • Data Sharing
  • Global Market Opportunity
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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