International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Pharmacology and Toxicology

Pharmacology and Toxicology

Due to the unique characteristics of rare diseases, developing medications for them might be difficult. The rarity of a condition makes drug development difficult. Due to a paucity of people, well-designed clinical trials to demonstrate efficacy can be difficult to conduct, and studies substantial enough to determine major hazards are nearly impossible to do. When no other options are available, patients may be ready to risk side effects in exchange for potential benefits, but the benefit-to-harm balance is difficult to quantify and may be unfavourable in some situations. Clinical pharmacology, a translational discipline, is a critical component of drug development, including therapeutic development for rare diseases. Health administrators have financial challenges when it comes to innovative pharmaceuticals, particularly orphan drugs. Individual rare disorders impact a small number of people, yet they collectively affect a significant portion of the population. They require our assistance.

  • Drug Repurposing
  • Safety Assessment
  • Clinical Trials
  • Patient Involvement
  • Translational Research
  • Clinical Pharmacology
  • Orphan Drug Development
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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