Victor Shengkan jin, Rutgers University, United States
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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
Orphan Drugs 2023
Oral Presentation
Fereshteh Sedaghat, Aristotle University, Greece
The proteoglycan named heparan sulfate (HS) is found in the extracellular matrix and also on cell surface and may represent one of the most biologically important glycoconjugates, playing essential role in a variety of different events at molecular level. Heparanase is an endo-gl [....] » Read More
Keynote Presentation
Jiao Fuyong, Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Kawasaki disease,It is a systemic vasioinflammatory disease in children of Asian descent,Coronary artery injury is its major complication,The disease has now become a major cause of acquired heart disease in children.The etiology of Kawasaki disease is not fully clear, and its pa [....] » Read More
Title : What does a technology-enabled patient concierge mean to the orphan drugs industry?
Harsha Rajasimha, Jeeva Informatics Solutions Inc, United States
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Title : Improving regulatory confidence for formal early phase clinical development.
Candida Fratazzi, BBCR Consulting - Massachusetts, United States
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Title : Scleritis and systemic diseases: What should know the internist/rheumatologist ?
Philip Bielefeld, Avignon Hospital, France
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Title : The foundation for rare disease and its role in the european rare disease research landscape
Daniel Scherman, Fondation Maladies Rares, France
The French rare disease research and clinical field has been a pioneer by structuring itself through different action plans. Competence centres and Reference centres are organized within Rare Disease Health Networks (Filières de Santé Maladies Rares), which ha [....] » Read More
Title : Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy
Magali Taiel, GenSight Biologics, France
Leber hereditary optic neuropathy (LHON) is a rare, maternally inherited mitochondrial genetic disease with a continued high unmet medical need. Three primary point mutations in the mtDNA are responsible for LHON in approximately 90% of subjects: G3460A, G11778A and T14484C, loca [....] » Read More
Title : Autoimmunity to the autonomic nervous system-The clue for many clinical entities
Yehuda Shoenfeld, Ariel University, Israel
Covid-19 virus is an autoimmune virus and it is more notorious than EBV. It induces autoimmune diseases by hyper-stimulation of the immune system combined in molecular mimicry. Post COVID-19 Syndrome (PCS) is a complex of various symptoms developing a month or more [....] » Read More
Title : Should PLENADREN ®, a modified release oral hydrocortisone preparation for glucocorticoid substitution, any longer be an „Orphan Drug“ ?
Wolfgang K.H. Oelkers, Freie Universität Berlin, Germany
Plenadren ( 20 and 5 mg tablets of modified release hydrocortisone (HC)), was developed for substituting patients with primary (prim) and secondary (sec) Adrenal Insufficiency (AI) with a single morning glucocorticoid (GC) dose. The orphan status was based on a single publication [....] » Read More
Title : Leveraging Data to Accelerate Rare Disease Treatment and Drug Development
Craig Martin, Global Genes, United States
Short Description of what will be discussed during the presentation (about 250 - 500 words) Patient data is perhaps the most valuable asset rare disease communities can leverage to generate research interest and spur drug development. Initiatives to drive the collect [....] » Read More