Pat Furlong

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands

Rotem Orbach, Monica Rebeca Gil Garzon, Hildegard Büning, Carsten G. Bönnemann et autres

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Transforming Objective Participatory Patient Advocacy

Pat Furlong, Timothy R. Franson, Peter J. Pitts

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A Parent Project Muscular Dystrophy-sponsored International Workshop Report on Endocrine and Bone Issues in Patients with Duchenne Muscular Dystrophy: An Ever-changing Landscape

Leanne Marie Ward, David R. Weber, Sze Choong Wong, Susan D. Apkon et autres

In April 2023, over 30 experts and advocates from four countries met in Rome, Italy to discuss unmet needs in endocrine and bone health care for individuals with Duchenne muscular dystrophy (DMD). Despite recent advances in muscle-targeted therapy, long-term glucocorticoids (GC) remain …

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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint

Laurent Servais, Paul Strijbos, Margaux Poleur, Andrada Mirea et autres

Stride velocity 95th centile (SV95C) is a wearable-derived endpoint representing the 5% fastest strides taken during everyday living. In July 2023, SV95C received European Medicines Agency (EMA) qualification for use as a primary endpoint in trials of patients with Duchenne muscular dystrophy …

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A matrix tool to foster patient engagement in children, adolescents and young adults: report from a multistakeholder workshop

Maria Cavaller-Bellaubi, Eva Degraeuwe, Johan Vande Walle, Elke Gasthuys et autres

Patient engagement has become increasingly important in drug development, yet structured guidance to engage children (2–11 years old), adolescents (12–17 years old), and young adults (18–24 years old) is lacking. This study aims to create a matrix tool to foster paediatric patient …

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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Ryan A. Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard et autres

Purpose: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). The objective of this study was to identify how value assessment …

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Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease

Craig M. McDonald, Eric Camino, Rafael Escandon, Richard S. Finkel et autres

Background: Duchenne muscular dystrophy (DMD) and related dystrophinopathies are neuromuscular conditions with great unmet medical needs that require the development of effective medical treatments. Objective: To aid sponsors in clinical development of drugs and therapeutic biological products for treating DMD across the …

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Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022

Kim Phung, Nicola J. Crabtree, Anne M. Connolly, Pat Furlong et autres

Individuals living with Duchenne muscular dystrophy (DMD) are at significant risk of bone fragility due to osteoporosis, with the most potent drivers of fragility fractures in this context stemming from the aggressive myopathy and long term oral glucocorticoid therapy. Young people with …

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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies

Ryan A. Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard et autres

ABSTRACT BACKGROUND Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). OBJECTIVE To identify how value assessment frameworks may need to …

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Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Craig M. McDonald, Eduardo Marbán, Suzanne B. Hendrix, Nathaniel J. Hogan et autres

BACKGROUND: Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy. METHODS: CDCs) or placebo …

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Mincing Words

Pat Furlong, Sheila C. Moeschen

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Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

Hannah Staunton, Claire Trennery, Rob Arbuckle, Maitea Guridi et autres

BACKGROUND: In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations. Thus, there is a need …

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