International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Revolutionary Changes

Revolutionary Changes

Regrettably, "rare disease" has historically been low on the public health agenda, a fact that the 350 million individuals worldwide who suffer from these diseases are well aware of. This is unsurprising since, while each of these rare diseases has too little a health burden to make a simple health-economic justification, rare diseases are thought to be just as common as other disorders. Rare disease detection and management is an example of an area that has profited from technological advancements. The integration of new medicines for rare diseases into existing care systems, on the other hand, calls into question a translational medicine premise that there is a linear pathway from bench to bedside. In awareness of the impact of patterns of clinical interventions and associated health services on patients' and their families' quality of life, a range of patient-oriented outcomes may be most relevant to examine for incremental therapies.

  • Recent Trends
  • Novel Innovations
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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