The call to treat rare diseases has become increasingly greater around the world than ever before. Supportive organisations including patient groups, national health authorities, health trusts, academia, HTAs, pharmaceutical and biotechnical companies are now progressively growing in numbers and sophistication in their approaches to medicinal care for those with rare and ultra-rare diseases.

SMi’s Orphan Drugs & Rare Diseases conference will present briefings from key opinion leaders, those with hands-on experience of regulating new drug discoveries, companies that have already developed advanced orphan drugs & successfully been granted orphan medicine status and selected experts in the field. Our expert speakers will offer unique insights into cutting-edge international developments taking place in: expanding the reach of medicine to previously untreatable and unreachable patients with rare diseases; different regulatory and policy environments; new drug discoveries; innovative business strategies & funding & financing models, and the importance of partnerships with patient groups and those at the point-of-care.

Reasons why you should attend:
• Network with key decision makers at leading companies including GSK Rare Diseases, Novartis and Shire
• Evaluate innovations in virus-mediated gene transfer and transgene expression
• Utilise successful partnering strategies and economic models to ensure international market entry
• Obtain funding for research and development
• Maximise your collaborations with HTA assessors and patient groups

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