Dear Colleagues, Partners, Researchers, Physicians, Entrepreneurs, Business leaders and Friends

As a member of the Organizing Committee of the 2nd International Conference on Orphan Drugs and Rare Diseases (Orphan Drugs-2025) to be hold in Rome, Italy, in March 13-15, 2025, it is my genuine pleasure to welcome you to this important event and this unique City being the Grand Symbol of wealth, prosperity, luxury, and exclusivity! And full of History, Culture, Science and Art as well!

Advances in fundamental, translational and clinical research as well as in environmental sciences and the availability of biomarkers are beginning to transform the healthcare landscape. Biomarker and target-driven platforms go into bringing the most promising experimental therapies, diagnostic, predictive, prognostic, and monitoring technologies to the clinic. Orphan Drugs-2025 does serve as a trans-disciplinary biodesign-inspired platform for experts, stakeholders, and advocates to come together to discuss advancements, challenges, and opportunities in the field. The Event will prompt a permanent information stream between basic researchers & biodesigners, clinical practitioners, healthcare providers and policy-makers to perform adequate regulations and creation of new guidelines to advance healthcare-related science, technologies and implementation in daily practice of orphan and rare diseases. The local community is aiming to bring together solution providers, biotech companies, clinical researchers, regulatory professionals and charity leaders together to discuss possible ways to accelerative orphan drug development and access to rare disease patients, including the introduction of recent technologies and products to help aid the access of the resources of personalized & precision medicine.

This Conference will introduce the esteemed professionals and experts in rare and orphan diseases from around the world to discuss the new developments and breakthroughs in the topical area of research and practice to engage in meaningful discourse and exchange valuable insights. The Conference may cover a wide range of topics related to rare and orphan diseases, including:

(i) landscape on orphan & rare diseases

(ii) rare nutritional and metabolic diseases;

(iii) mosaicism in orphan and rare diseases: biomarker-driven diagnostic, predictive, prognostic and screening approaches towards monitoring of orphan and rare diseases;

(iv) genetic counselling of orphan and rare diseases;

(v) drug discovery, development and design: the innovations in rare and orphan disease therapeutics;

(vi) patient-centered care and transdisciplinary healthcare team;

(vii) molecular epidemiology and biostatistics;

(viii) patient-related advocacy, support, and empowerment;

(ix) building a supportive ecosystem for rare and orphan diseases.
 

This interactive meeting fosters knowledge exchange among clinicians, clinical scientists, researchers and bioinformaticians working on rare diseases. The Conference would generate solution-focused approaches surrounding patient access, the latest innovations, reimbursement, commercialization, launch, and more as we do unify in areas of rare and orphan diseases-related medical need.

Personally I am proud to be involved into this auspicious event where a large number of world’s leading scientists, researchers, clinicians, industrialists and academicians will be exchanging new scientific ideas and sharing their innovative research and experiences in their respective fields.