Chiesi Pharma Ltd

We're delighted that Chiesi Pharma Ltd 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 Pharma Ltd

In 2020, Chiesi launched Chiesi Global Rare Diseases, a business unit focused on supporting people living with rare diseases. The Global Rare Diseases unit works in collaboration with the wider Chiesi Group to harness the full resources and capabilities of our global network. Our goal is to support as many people as possible living with rare diseases, so that they can lead the lives they want. We collaborate with the rare disease community to bring the voices of those affected to the fore. 

Amicus Therapeutics

Company overview to follow...
                                                                                                    

Immedica

Headquartered in Stockholm, Sweden, Immedica is a pharmaceutical company focused on the commericalisation of medicines for rare and specialty diseases. Our services cover marketing and sales, compliance, pharmacovigilance, compliance, quality assurance, regulatory and medical affairs and market access. Serving patients in more than 50 countries, we are fully dedicated to helping those living with diseases which have a large unmet medical need. Our therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care.

Orchard Theraputics

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.

Aspire Pharma Ltd

Company overview to follow...
                                                                                                    

We are developing new treatment options to help patients with rare metabolic disorders. Azafaros was founded in 2018 by a team of experienced industry professionals and scientists aspiring to address rare genetic metabolic disorders through a pipeline of oral small molecules with disease-modifying potential. Based on discoveries from Leiden University and Amsterdam UMC, Azafaros’s proprietary lead compound, nizubaglustat, will initially address inherited life-threatening lysosomal storage diseases for which there are no effective therapies. This orally available azasugar compound interferes with the metabolism of glycolipids and uniquely affects several key disease pathways through a dual mode of action. Leveraging the know-how of its team and partners in orphan drug development, the company is advancing its lead program in the clinic while further expanding its product pipeline into other rare metabolic diseases through its drug discovery efforts.

BioMarin

Company overview to follow...


                                                                                                    

HealthNet Homecare

Company overview to follow...
                                                                                                      

JCR Pharma

Company overview to follow...





                                                                                                     

Logixx Pharma Ltd

LogixX Pharma is a speciality pharmaceutical company based in Berkshire, United Kingdom. LogixX Pharma was founded in 2010 and is an independent privately owned entrepreneurial and dynamic enterprise - with core competencies in the fields of Pharmaceuticals, Medical Devices and OTCs. 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.

Nutricia Amn Ltd

Company overview to follow...
                                                                                                    

Travere Therapeutics

Company overview to follow...
                                                                                                      

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 are the leading organisation for Inherited Metabolic Disorders (IMDs), supporting thousands of people worldwide through providing individual support, building communities, and continually advocating for and empowering those living with IMDs. Since its inception in 1981, our organisation has strived to support those in the rare community, expanding newborn screening, accelerating the development of orphan drugs, and acting instrumentally in establishing EURORDIS, ensuring people living with IMDs have the best quality of life possible. Moving forward, utilising our 10 year strategy we will continue to improve the lives of people living with Inherited Metabolic Disorders, and to work collaboratively with our key partners and supporters to ensure that we achieve this. Our strategy focuses on four main pillars: Individual Support Building Communities Empowerment Advocacy

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.