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.

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

FSHD Global 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

Hub Leadership

The Hub leadership team have extensive experience in neuromuscular diseases, clinical services, research, and patient engagement — ensuring a robust foundation for mission.

Ken Kahtava, General Manager
Ken Kahtava

GENERAL MANAGER

Ken is Chief Operating Officer of the FSHD Society and co-founder of the Hub. A serial technology and business entrepreneur, Ken is also a veteran leader in the nonprofit research industry with more than twenty years’ experience in establishing public-private partnerships in multiple rare disease communities to accelerate research and access. Ken leads the Hub team and our business development efforts across the global life sciences sector.

Lawrence Korngut, Chief Medical Officer
Lawrence Korngut, MD MSc FRCPC

CHIEF MEDICAL OFFICER

Lawrence is a clinician investigator at the University of Calgary where he conducts research in registry best practices at the Korngut Registry Science Lab. He led the development of the Canadian Neuromuscular Disease Registry which includes 48 participating clinics across Canada which has successfully recruited over 6000 patients across four disease sub registries. Lawrence is a recognized key opinion leader in FSHD and co-Founder of Lumiio.

Lauren Morgenworth, Director of Clinical Services
Lauren Morgenroth

DIRECTOR OF CLINICAL SERVICES

Lauren led the development of TRiNDS starting in 2016. Prior to that in 2004, she joined the Cooperative International Neuromuscular Research Group (CINRG), an academic clinical trial consortium for which TRiNDS is also the Coordinating Center. Lauren is a genetic counselor and worked in both pediatric and adult muscular dystrophy associations clinics. Lauren is CEO of TRiNDS.

Victoria Hodgkinson, Chief Scientific Officer
Victoria Hodgkinson, PhD

CHIEF SCIENTIFIC OFFICER

Victoria is CSO at Lumiio and the FSHD Global Innovation Hub. A recognized global expert in registries following her work on large-scale international projects such as the Global SMA Registry and the Global Acromegaly Registry. Her experience in registries includes dataset derivation, protocol and ethics, privacy legislation, and platform design. Victoria also has extensive and varied experience in data analysis and publishing findings utilizing real-world registry data. Victoria co-leads the implementation of platform user requirements in addition to leading ongoing registry projects for clients.

Blaine Penny, Director
Blaine Penny

DIRECTOR, INNOVATION AND RWD/E

Blaine is CEO of Lumiio and leads project management for the FSHD Global Innovation Hub. He has over 20 years of experience in the health, technology, and engineering sectors leading and managing large teams and portfolios, capital investment projects, product management, M&A, and go-to-market strategies. Fifteen of those years were in Executive roles and included Co-founder and CEO of MitoCanada, CEO of the start-up engineering firm Integrated Sustainability, and Vice President of the global technology firm IHS Markit.

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.

Neil Camarta, Director
Neil Camarta

Neil is co-founder of FSHD Canada Foundation. Neil has held senior leadership positions with Shell, Petro-Canada, and Suncor. He has extensive experience in the oil and gas industry in Canada and globally. He is currently leading two new start-up companies working on cheaper and cleaner technologies for the energy industry. Neil holds a degree in Chemical Engineering from the University of Alberta.

Stuart Lai, Director
Stuart Lai

Stuart is a board member of the FSHD Society and a serial technology entrepreneur. A technology architect and software engineer, Stuart has many decades of experience leveraging technology and data to drive business outcomes in various industries.

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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