Insights

What is FSHD?

Facioscapulohumeral muscular dystrophy, or FSHD, is a genetic disorder that leads to the relentless weakening of skeletal muscles.

Typically beginning in early teenage years with the loss of muscles in the face (facio), shoulders (scapula), upper arms (humerus), legs or core, FSHD can spread to any muscle. Around 20 percent will need a wheelchair by age 50. Over 70 percent experience debilitating pain and fatigue. FSHD is estimated to affect nearly one million people worldwide.

For more information about FSHD, go to fshdsociety.org/what-is-fshd .

About Us

Our mission is to accelerate the development of effective FSHD therapeutics and get treatments to people faster.

Who We Are

Global FSHD Innovation Hub is a wholly-owned subsidiary of the FSHD Society’s 501(c)3 founded in 1991. The Hub is a commercial Limited-Liability Company (LLC) that contracts with biopharma companies to accelerate their product development lifecycle. It operates as a partnership entity to streamline client engagement and contracting processes.

Hub Delivery Partners

A full service CRO specializing exclusively in neuromuscular disorders

a scientific, clinical, digital technology and insights platform and services company focused on accelerating health outcomes and value in drug development

the world’s largest research-focused advocacy organization with operations across 5 continents

Solutions

  • The Hub serves as a single end-to-end partner for biopharma companies to accelerate their products in FSHD.
  • Our offerings are purpose-built and proven — exclusively for FSHD, making us the only such partner in the world providing proven and innovative solutions along the FSHD product lifecycle.

Solutions

Our FSHD Therapeutic Advisory Group (TAG) is a multi-disciplinary group of international experts that provides confidential review and evaluation of FSHD therapeutics for companies looking to optimize their design, development and fit for FSHD. The TAG is comprised of academic and industry drug development experts, representatives of patient advocacy groups, KOL’s, and regulatory bodies.

  • Engaging well-characterized FSHD patients and relevant clinical trials together is a key challenge in FSHD research. The Hub facilitates this on behalf of our biopharma industry clients.
  • The Hub provides strategic guidance and superior implementation of FSHD data programs to bridge clinical development and post market real-world evidence needs to drive global outcomes and accelerate time to market. From data insights for designing and executing successful FSHD clinical trials, to meaningfully engaging the FSHD community, to expanding market access, our experts and tools help pharmaceutical companies overcome barriers to rapidly realize results.
  • The Hub provides specialized contract research organization (CRO) services – solely dedicated to conducting high quality clinical trials in FSHD.
  • We offer expert dedicated solutions in FSHD trial management, clinical monitoring, site management, data management, study outcome measures, and other clinical research services. We are uniquely focused on FSHD and our expertise has been drawn from academic FSHD clinical trial networks.
  • We are experts in site efficiency and work directly with site staff to improve their operational effectiveness, removing barriers and obstacles and ensuring excellence. For our biopharma clients, this means faster contracting and site start up.

Solutions

  • FSHD community readiness is core to the Hub’s mission. Ensuring patients and healthcare providers are fully informed about FSHD and available treatments is critical to delivering on our promise to accelerate patient access to treatments
  • Our FSHD Community Navigator Platform is a global advocacy platform operating on 5 continents and dozens of countries. It is comprised of the tools and resources needed to drive education, KOL engagement, regulatory and access advocacy.
  • Biopharma companies wanting to introduce new FSHD treatments in many countries are asked to submit dossiers providing evidence for clinical and cost effectiveness of the new technology to Health Technology Assessments (HTA) agencies and payers. Developing evidence for HTA dossiers is particularly challenging in FSHD given the sparsity of available evidence. The Hub maintains a disease-level HTA package for biopharma companies to accelerate their FSHD dossier development.

Solutions

  • The Hub serves as a single end-to-end partner for biopharma companies to accelerate their products in FSHD
  • Our offerings are purpose-built and proven — exclusively for FSHD, making us the only such partner in the world providing proven and innovative solutions along the FSHD product lifecycle.

Our FSHD Therapeutic Advisory Group (TAG) is a multi-disciplinary group of international experts that provides confidential review and evaluation of FSHD therapeutics for companies looking to optimize their design, development and fit for FSHD. The TAG is comprised of academic and industry drug development experts, representatives of patient advocacy groups, KOL’s, and regulatory bodies.

  • Engaging well-characterized FSHD patients and relevant clinical trials together is a key challenge in FSHD research. The Hub facilitates this on behalf of our biopharma industry clients.
  • The Hub provides strategic guidance and superior implementation of FSHD data programs to bridge clinical development and post market real-world evidence needs to drive global outcomes and accelerate time to market. From data insights for designing and executing successful FSHD clinical trials, to meaningfully engaging the FSHD community, to expanding market access, our experts and tools help pharmaceutical companies overcome barriers to rapidly realize results.
  • The Hub provides specialized contract research organization (CRO) services – solely dedicated to conducting high quality clinical trials in FSHD.
  • We offer expert dedicated solutions in FSHD trial management, clinical monitoring, site management, data management, study outcome measures, and other clinical research services. We are uniquely focused on FSHD and our expertise has been drawn from academic FSHD clinical trial networks.
  • We are experts in site efficiency and work directly with site staff to improve their operational effectiveness, removing barriers and obstacles and ensuring excellence. For our biopharma clients, this means faster contracting and site start up.
  • FSHD community readiness is core to the Hub’s mission. Ensuring patients and healthcare providers are fully informed about FSHD and available treatments is critical to delivering on our promise to accelerate patient access to treatments
  • Our FSHD Community Navigator Platform is a global advocacy platform operating on 5 continents and dozens of countries. It is comprised of the tools and resources needed to drive education, KOL engagement, regulatory and access advocacy.
  • Biopharma companies wanting to introduce new FSHD treatments in many countries are asked to submit dossiers providing evidence for clinical and cost effectiveness of the new technology to Health Technology Assessments (HTA) agencies and payers. Developing evidence for HTA dossiers is particularly challenging in FSHD given the sparsity of available evidence. The Hub maintains a disease-level HTA package for biopharma companies to accelerate their FSHD dossier development.

Hub Board of Directors

Mark Stone, Director
Mark Stone

CEO of FSHD Society and has served as an executive leader of research-focused patient advocacy nonprofit organizations in multiple rare diseases since 2004. Mark has launched drug discovery initiatives anchored by clinical trial networks globally to expedite potential treatments across multiple diseases communities.

Mel Hayes

Mel Hayes has over three decades of executive leadership experience in building and leading successful commercial and development organizations at major pharmaceutical and biotech companies including Bioverativ/Sanofi, and Fulcrum Therapeutics. With a strong record in rare disease launches and corporate strategy, he brings valuable expertise in advocacy and commercialization that supports the Hub's ability to translate research into real-world impact. 

Hans Van Bylen

Hans Van Bylen is a global business leader with more than three decades of executive experience in the consumer goods and chemical industries. A former CEO of Henkel, he led major acquisitions and strategic growth initiatives that expanded the company's international reach. He is currently Chairman of Ontex and Etex and a board member of Lanxess and AkzoNobel. His leadership and global perspective will strengthen the Hub's mission to advance FSHD drug development.

Neil Camarta, Director
Neil Camarta

Neil Camarta is a chemical engineer and member of the Canadian Academy of Engineering who has held senior leadership positions across the oil and gas industry. He co-founded Western Hydrogen and Enlighten Innovations, two cleantech start-ups focused on green fuel and grid-scale battery technologies. He also co-founded the FSHD Canada Foundation, Solve FSHD, and Project Mercury, and brings extensive knowledge in driving global progress in FSHD research.

Stuart Lai, Director
Stuart Lai

Stuart Lai brings more than 30 years of experience in software engineering and data infrastructure from leading financial and technology firms, including Goldman Sachs, Refinitiv, and Crux Informatics. A computer and electrical engineer, he has built large-scale systems that support global analytics and data access. As Chair of the FSHD Society's Patient Access and Advocacy Committee, and a new member of the Board of the Global FSHD Innovation Hub, Lai combines his deep technical expertise with a personal commitment to improving access and outcomes for people living with FSHD.

Why the Innovation Hub Was Created

The Hub serves as a comprehensive end-to-end partner for biopharma companies, providing innovative solutions along the FSHD product lifecycle that solve key challenges in FSHD:

Poor FSHD trial capacity

Multiple promising therapies for FSHD are in development but there is a lack of capacity in the FSHD research community due to:

  • Lack of operational experience and excellence at sites
  • Wide gap between best sites and average sites in optimized capacity and delivery on trial commitments
  • Lack of landscape and feasibility understanding to identify qualified early phase trial sites with trained expertise 
  • Slow site contracting process resulting in trial start-up delays
  • Delays in transition from early phase to pivotal trial

Many patients are unable to access approved therapies

It is expected that many patients globally will not have access. Payers in many countries will implement rigid and exclusive reimbursement criteria. Overcoming this means we must address:

  • Siloed patient registries and data with lack of operational excellence and experience resulting in limited accessibility and completeness of data
  • Lack of patient population data and disease underdiagnosed
  • Education — disease state not well understood by clinical and patient community
  • Lack of post-marketing real-world data (RWD) to support innovative risk sharing and reimbursement

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