Thank you to our Sponsors and Exhibitors - this event is made possible by their support.
None of the supporting organisation have influenced the event content or choice of speakers.
Chiesi Global Rare Diseases
We're delighted that Chiesi Global Rare Diseases are supporting the Pre-meeting and also our Platinum supporter (more information below).
Immedica
We're delighted that Immedica are supporting the Pre-meeting and also one of our Gold supporters (more information below).
Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit https://www.chiesi.uk.com/rare-diseases
Orchard Theraputics
Orchard Therapeutics is dedicated to empowering life and inspiring new possibilities for people affected by certain rare inherited diseases. Through our mission to develop one-time, disease altering therapies for a range of devastating genetic conditions, we seek to have a lasting effect on children, families, caregivers, and society.
Immedica
Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialisation of medicines for rare diseases.Serving patients in more than 50 countries, Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need.Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care.
Amicus Therapeutics
Company overview to follow...
Azafaros B.v.
Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors including Forbion, Jeito Capital, Seroba, Pictet Group, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.
HealthNet Homecare
HealthNet is a trusted provider of advanced homecare services, specialising in the delivery of complex therapies for patients with rare, orphan, and highly specialised conditions. With deep expertise across high-cost and low-volume treatments, we support NHS partners, clinicians, and patients by safely transitioning complex care from hospital to home.Our High Tech Nursing services are built on a strong foundation of clinical governance, specialist training, and national nursing capacity. We deliver complex treatment pathways - including biologics, injectables, and advanced therapies - with precision, consistency, and patient-centred care.Digitally enabled and clinically led, our approach ensures full visibility, safety, and adherence throughout the treatment journey.At HealthNet, we combine clinical excellence with compassionate care to improve access, outcomes, and patient experience in even the most complex therapy areas.
Logixx Pharma Ltd
LogixX Pharma is a speciality pharmaceutical company based in Berkshire, United Kingdom.As an innovation company we specialise in developing and commercialising Products and Services from Research and Development projects. The company and has quickly established itself in Europe by acquiring the rights to market and distribute innovative Pharmaceutical, Medical Device and OTC products.
Lucane Pharma
Lucane Pharma is specialised in the development and commercialisation of medicines targeting rare metabolic disorders. Lucane Pharma aims to develop medicines for patients with urea cycle disorders (UCD) and accordingly provide patients suffering from rare metabolic disorders a real improvement in the management of their disease. • We improve the formulations of existing products. • We develop new presentations fulfilling a specific need. • We improve the accessibility of our products worldwide and for a fair price. Customer engagement is what drives us. We step in quickly, are entrepreneurial and decisive. We are accountable for our actions and operate transparently. We believe that medicinal products must be readily available for patients and also be obtainable at a reasonable price. If we can help a doctor to quickly get a crucial drug to a patient via a pharmacist, this is our highest priority. Thomas Calis, Managing Director.
Meta Healthcare
Company strategy: We work with healthcare experts in metabolic disease to bring to market innovative product presentations that offer a point-of-difference to help overcome unmet clinical needs, enhance patient experience and, ultimately improve patient outcomes.
Company goal: To become the UK’s leading metabolic pharma company delivering a complete and wide range of low protein foods/supplements and medicines.
Nutricia Amn Ltd
Nutricia has over 70 years of experience in providing essential nutrition and services to support adherence to metabolic diets at every stage of life. We believe in the power of nutrition to make a positive difference to health. This belief drives our commitment to pioneer life-saving and life-changing nutritional solutions. We work in close partnership with parents, patients, carers, and healthcare professionals. Through passion and the best-in-class science, we bring health through nutrition to as many people as possible.For many patients diagnosed with Inherited Metabolic Disorders (IMDs), a severely restricted diet, often for life, is essential to optimise outcomes. Our comprehensive portfolio includes effective and proven nutritional solutions for IMDs, catering to every life stage with our extensive product ranges. All our products are designed to meet the changing needs in taste, formats, and intake volume, ensuring that patients receive the right balance of nutrients at the right time.We also offer a variety of low-protein foods under our Loprofin range, including baking mixes, pasta, and rice, to support patients who need to follow a low-protein diet. Our commitment extends to providing services for patients that make life easier, focusing on education, cooking, and product ordering, to help them and their carers manage their dietary needs more effectively.
Visit our stand to discover more about our innovative products and services, and learn how Nutricia can support you and your patients!
Nutrinovo
Nutrinovo are a UK clinical nutrition company with one aim at its core – to make ‘simply innovative nutrition’ available to patients through the health professionals that manage their care and support. We are dedicated to providing high-quality nutritional solutions that address a range of clinical needs.
Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and potentially life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the centre of our ambitions.
UCB
At UCB, we believe that everyone deserves to live the best life that they can. That’s why – as a global biopharmaceutical leader – we’re focused on creating valuable solutions that make improvements to the lives of people living with neurological and autoimmune conditions. We work closely and forge strong connections with patients, caregivers, healthcare professionals and other stakeholders who know the challenges of these diseases. We bring together the expertise, talent, tools and scientific ingenuity needed to pursue what’s right for those who live with severe diseases.Inspired by patients, driven by science.
Ultragenyx
Ultragenyx is a biopharmaceutical company focused on the development of treatments for serious rare and ultrarare genetic diseases. The company aims to address conditions with high unmet medical need and well-understood biology, where there are typically limited or no treatment options available. Ultragenyx is led by a team with extensive experience in rare disease research and development. Its approach is based on time- and cost-efficient drug development processes to support the advancement of potential treatments.
Vitaflo
Company overview to follow...
Metabolic Support UK
Metabolic Support UK is the umbrella patient organisation for people living with and affected by Inherited Metabolic Disorders (IMDs). We are a UK-based organisation with an international reach.We enable our inherited metabolic disorder community to live well by offering individual support, building connections, and developing evidence-based insight.
LSD Collaborative
The UK LSD Patient Collaborative Group is comprised of patient organisations representing those affected by Lysosomal Storage Disorders (LSDs). We have joined together to form an action group to advocate and work on behalf of LSD patients and their families in the UK. The group is made up of representatives from 7 charities in the UK who provide support and information to patients and families affected by different LSDs. Together we represent over 70 rare and ultra rare diseases and almost 2500 patients and their families.
The Lily Foundation
The Lily Foundation is the UK’s largest mitochondrial disease charity, founded in 2007 by Liz Curtis in memory of her daughter Lily, who died from mitochondrial disease at just eight months old. The charity’s vision is for a world in which every mitochondrial disease patient has a voice and access to treatment, support to improve their life and, ultimately, a cure.
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