Orphan Europe, part of the Recordati Group, is a pharmaceutical company aiming at providing treatment for patients with unmet medical needs suffering from rare diseases. Since 1990, Orphan Europe is established as one of the most active players in the field of rare diseases.
Orphan Europe is committed to the improvement of knowledge about rare diseases among healthcare professionals. The best example is the support of Recordati Rare Diseases Foundation that provides unique, independent and high-level education on rare diseases.
Orphan Europe shares the conviction that every single patient has the right to the best possible treatment. For more information, please visit www.orphan-europe.com
Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from rare disorders. OT-58, our lead drug development candidate, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases. For more information, please visit www.orphantechnologies.com.
OT-58, Orphan Technologies’ lead drug candidate, is a modified recombinant enzyme replacement therapy in development as an enzyme replacement therapy for patients suffering from the rare disease classical homocystinuria. Classical homocystinuria is a genetic metabolic disease caused by a deficiency in the CBS enzyme leading to elevated levels of the amino acid homocysteine. OT-58 has consistently demonstrated significant reductions in homocysteine levels across multiple models of homocystinuria and has the potential to improve metabolic control, reduce or remove dietary restrictions, and positively impact clinical outcomes. OT-58 is anticipated to enter clinical evaluation in 2018 and has been granted Orphan Status by the US Food and Drug Administration and EMA. In addition, based on its mechanism of action, OT-58 has therapeutic potential in other diseases.
Bio for Nutricia Metabolics coming soon.
100 years by your side
Founded in Genoa in 1915 by Edoardo Maragliano, the man who discovered the first tuberculosis vaccine, PIAM is an Italian pharmaceutical company that has developed its core business in specialist and ethically significant therapeutic areas with prescription drugs.
Over time it has gained particular expertise in hereditary metabolic diseases. In the 70s, PIAM pioneered a specialist nutritional line to support its pharmaceutical products. It is able to offer a complete response for patients with disorders requiring particular nutritional therapy, throughout every phase of their lives.
Here at Piam, our priorities are the different needs faced by people with unusual or rare disorders.
We want to continue being the preferred partner for everyone who suffers from these diseases.
We offer effective solutions and promote a health-centric culture focusing on individuals and their requirements.
Our approach to care derives from more than 100 years of experience alongside doctors and the scientific community.
Thanks to our exclusive history and expertise, we are able to develop our offer around tangible needs faced by doctors, patients and caregivers, providing them with the necessary treatments, whether pharmacological or nutritional, as well as services to help with the effective management of their specific needs.
Our expertise is the result of more than a century of involvement in the pharmaceutical sector and thirty years of research into nutritional therapy for rare diseases.
By combining nutritional and pharmacological therapies, we are able to offer physicians who consistently chose us a complete platform of integrated therapeutic solutions and services for customised care.
Through a solid network constructed over time with high-profile international scientific partners, as well as targeted clinical research and development, we are able to seize the best and most innovative therapeutic opportunities in terms of efficacy and safety available on the world market.
We adopt an approach that enables us to provide the most appropriate pharmacological therapy solutions for unmet needs as quickly as possible, via a flexible, yet ethically and scientifically stringent business model.
We have had a single mission for more than a century, which looks set to remain the same for the next 100 years: commitment to providing appropriate and cutting-edge responses to growing health needs. For more information, please visit www.piamfarmaceutici.com/en/.
BioElectron is a platform biotechnology company that is using its expertise in redox chemistry to develop first-in-class therapeutics for CNS and non-CNS diseases characterized by redox defects. BioElectron’s lead clinical compound—EPI-743—is in late clinical stage development for mitochondrial disease and related orphan disorders with shared biochemistry, including cobalamin C defect. In addition, BioElectron has a rich pipeline of platform-derived compounds in various stages of development from preclinical to phase 2. For more information, please visit www.bioelectron.com
Vitaflo are at the forefront of developing innovative specialised clinical nutrition products for Metabolic Disorders such as Homocystinuria and other specific conditions. Our aim is to create products that combine the best of cutting-edge research with the lifestyle demands of modern living, ensuring the most acceptable products are available for the patient. By constantly evolving to meet patient needs, Vitaflo will continue to develop products which offer patients choice and help support them in complying with restrictive therapeutic diets. For more information, please visit www.vitafloweb.com.
Bio for MetaX is coming soon. For more information visit www.metax.org.
The aim of the Society is to foster the study of inherited metabolic disorders and related topics. The Society, founded in 1963, exists to promote the exchange of ideas between professional workers in different disciplines who are interested in inherited metabolic disease. Pursuing this aim by arranging scientific meetings, publications and in other ways considered appropriate by the Council. The Council is also supported by advisory council members who provide advice and meet with the Council at the annual symposium.
The Society, a registered charity, accepts donations from sponsors sympathetic to its aims. The Council, within its financial resources, is willing to spread interest and study in inborn errors around the world by selectively supporting membership and attendance at conferences from areas of the world where financial resources are more limited. Not funding research, but offering on request, advice to other organisations who do. The Society is also a limited company and the liability of members in the event that the Society is wound up is limited to £1 per member. For more information, please visit www.ssiem.org.
Cambrooke Therapeutics (expansion of Cambrooke Foods) was founded in 2000 by Lynn and David Paolella, the parents of two children diagnosed with a rare disease called phenylketonuria (PKU). PKU is one of the few genetic diseases, which is managed almost entirely with nutritional intervention. The Paolellas’ goal in forming Cambrooke was simple – to develop improved nutritional therapeutic options for those with serious medical disorders.
Today, Cambrooke produces medical formulas and foods for the management of a variety of medical conditions and we are continually innovating new nutritional options targeted at a wide array of diseases.
Leaders in Therapeutic Medical Nutrition
- Cambrooke was the first medical foods company to launch a natural intact protein for the dietary management of phenylketonuria called Glytactin™ (modified glycomacropeptide) and Tylactin™ (modified glycomacropeptide) for the dietary management of Tyrosinemia.
- Cambrooke collaborates with academia and industry partners to transform early phase development projects in therapeutic nutrition into viable commercial products.
- Cambrooke employs scientific experts in the development, manufacturing and commercialization of nutritional therapeutics for patients with rare diseases.
- Cambrooke’s products are supported by clinical evidence for the nutritional management of medical needs before they are selected for commercialization.
- Cambrooke believes that both large and small patient populations are important when it comes to complex nutritional requirements.
For more information, please visit www.cambrooke.com