HYBRID EVENT: You can participate in person at London, UK or Virtually from your home or work.

International Conference On Orphan Drugs and Rare Diseases

August 21-23, 2023

August 21 -23, 2023 | London, UK

Rare Diseases and Ultra Rare Diseases

Rare Diseases and Ultra Rare Diseases

Interest in rare diseases has expanded in recent years, as evidenced by politicians' and health authorities' agendas, yet ultra-rare diseases still receive far too little attention. Although no legal classification of an "ultra-rare" disease has been created, the National Institute for Health and Care Excellence has informally designated this subcategory for medications having indications for disorders with a prevalence of less than one in per 50 000 people. The approach by which ultra-rare disease research should be conducted, as well as the question of quality, are both critical considerations. Although less stringent criteria may be used for orphan pharmaceuticals than for drugs treating more prevalent diseases, this should not be used as an excuse to deny people with rare diseases the best available treatment. When a patient is diagnosed with a rare or ultra-rare condition, having the correct physician and treatment plan is just as vital as having a support system.

  • Ultra-Rare Disease
  • Diagnosis
  • Drug Development
  • Caregivers’ Role
Committee Members
Speaker at Orphan Drugs and Rare Diseases 2023 -  Daniel Scherman

Daniel Scherman

Fondation Maladies Rares, France
Speaker at Orphan Drugs and Rare Diseases 2023 -  Jiao Fuyong

Jiao Fuyong

Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Speaker at Orphan Drugs and Rare Diseases 2023 -  Victor Shengkan jin

Victor Shengkan jin

Rutgers University, United States
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Candida Fratazzi

Candida Fratazzi

BBCR Consulting - Massachusetts, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Philip Bielefeld

Philip Bielefeld

Avignon Hospital, France
Speaker at Orphan Drugs and Rare Diseases 2023 - Harsha Rajasimha

Harsha Rajasimha

Jeeva Informatics Solutions Inc, United States

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