The 2nd annual World Orphan Drug Congress showcases drug development, new drug discovery and innovations related to rare diseases.

LYSOGENE is a clinical stage pioneering biotechnology company engaged in providing safe and efficient innovative gene therapy treatments for patients affected with severe neurological diseases. Founded in recent years, LYSOGENE’s business and economic model is based on avant-garde drug development methodologies focused on scientific and medical intelligence, regulatory streamlining, and genuine...

Since its inception in 1986, the company has grown both organically and via acquisitions. Shire focuses its portfolio on providing products for specialist physicians to prescribe in markets where the company can build a significant position. This has resulted in 2009 total revenues of US $3 billion. Shire has offices in 28 countries, and its medicines are distributed in over 50. The Company is...

AVI BioPharma is focused on the discovery and development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. Applying pioneering technologies developed and optimized by AVI, the Company is able to target a broad range of diseases and disorders through distinct RNA-based mechanisms of action. Unlike other RNA-based approaches, AVI's...

OXON, Dendrite and Chiltern have formed a unique and complementary collaboration to offer a comprehensive service for global patient registries. Together, this collaboration is able to offer unparalled strengths in epidemiology, statistics, pharmacovigilance, quality of life, health economics, electronic registries and project management. www.oxonepi.com www.e-dendrite.com www.chiltern.com

Centric Health Resources, Inc. is a nationwide, Patient-Centered Health Management organization, serving patients with rare, ultra-orphan disorders. Biotech and pharmaceutical manufacturers maximize value by aligning with Centric for streamlined and flexible service offerings that include reimbursement expertise, delivery of specialty pharmaceuticals, peer health coaching, and clinical support...

Clinigen focuses solely on the consultancy, management and implementation of Named Patient and Early Access solutions. Clinigen supports its customers with Global Early Access/Named Patient Programs across late stage clinical development, pre- launch, launch and product withdrawal sections of the pharmaceutical life cycle, supporting patient and physician access to drugs in countries where those ...

Cerebricon Ltd., a member of the Charles River Discovery Services family since 2009, is a leading contract research organization focused in the area of specialized and customized preclinical disease models of the Central Nervous System (CNS) and Peripheral Nervous System (PNS). Cerebricon’s preclinical modeling platform, which includes small animal models for neurology and psychiatry, biomarkers...

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focusing on the discovery, development and marketing of innovative pharmaceutical products for the treatment of orphan neuromuscular diseases. Santhera currently has three compounds in seven clinical-stage development programs. Santhera's vision is to become a leading specialty pharmaceutical company offering...

QCTR is an international, niche CRO specialising in neurology, psychiatry and orphan indications. We conduct proof of concept and pivotal phase II and III trials. Through our unique approach we can deliver patient recruitment to our clients in these notoriously difficult areas. QCTR will get you to Last Patient In in as timely a manner as we got you to the First. QCTR are the world's first CRO...

PSR is the leading expert in orphan drug development. We assist our clients to design and execute successful clinical development strategies for orphan indications. We are specialized in complex development clinical programs requiring innovative regulatory and clinical approaches. PSR has a strong track record in supporting orphan drug applications and we can facilitate obtaining scientific...

Cerebricon Ltd., a member of the Charles River Discovery Services family since 2009, is a leading contract research organization focused in the area of specialized and customized preclinical disease models of the Central Nervous System (CNS) and Peripheral Nervous System (PNS). Cerebricon’s preclinical modeling platform, which includes small animal models for neurology and psychiatry, biomarkers...