HERTFORDSHIRE, UK – April 15, 2025 – PHARMExcel, a leading UK-based Clinical Research Organisation (CRO), proudly supports global efforts to raise awareness for Fabry disease and other rare conditions, including Mucopolysaccharidoses (MPS) and lysosomal storage disorders. Our commitment to improving outcomes for patients living with rare diseases extends beyond awareness months, we’re striving to accelerate clinical research every day for the 200 million people affected with a rare disease.
In 2025, PHARMExcel is actively participating in several national and international events that support the rare disease community, foster collaboration, and promote innovation in research. Our chosen charity partner this year, Rare Beacon, plays a vital role in amplifying the voices of those living with rare diseases and improving access to research and care.
PHARMExcel is proud to support the following events in 2025:
- WORLD Symposium – February 2025, San Diego, USA
A premier global research conference focused on lysosomal diseases. - Rare Disease Research UK – 2nd Annual Conference – March 2025, Manchester, UK
A platform uniting UK researchers and stakeholders to accelerate rare disease breakthroughs. - Rare Beacon Cambridge Showcase – June 2025, Cambridge, UK
An engaging event highlighting innovative research, policy, and patient advocacy in the rare disease space. - MPS Society’s “MPS Matters” Conference – June 2025, UK
Dedicated to improving understanding and support for individuals with MPS and related diseases. - 2025 International Drug Repurposing Conference – September 2025, Amsterdam
Brings together researchers, regulators, and patient groups to explore novel uses for existing medicines in rare disease treatment. - World Orphan Drug Congress Europe – October 2025, Amsterdam
Europe’s leading conference for advancing orphan drug development and access to therapies.
“We’re proud to work alongside our partners, clients, and patient communities to drive meaningful change in rare disease research,” said Yvanne Enever, CEO and Founder of PHARMExcel. “From early-phase clinical trials to strategic regulatory guidance, our commitment to rare diseases, including Fabry and MPS, reflects our mission to deliver quality-driven, patient-focused research with impact.”
About Fabry Disease
Fabry disease is a rare genetic lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. This deficiency leads to the accumulation of a fatty substance called globotriaosylceramide in the body, resulting in pain, kidney failure, heart disease, and stroke. Symptoms vary widely and can begin in childhood or adulthood.
About Mucopolysaccharidoses (MPS)
MPS diseases are a group of inherited metabolic disorders caused by the body’s inability to produce specific enzymes. These conditions lead to progressive damage to the heart, bones, joints, respiratory system, and central nervous system. MPS conditions are complex, life-limiting, and currently have no cure.
About PHARMExcel
PHARMExcel is an award-winning CRO supporting the delivery of high-quality clinical research across the UK and beyond. With a strong focus on biotech innovation and rare diseases, PHARMExcel partners with academia, healthcare providers, and industry to accelerate treatments from bench to bedside.
