ATOM International provides functional testing training and oversight in the support of rare neuromuscular disorders.
Our global team of physiotherapists help ensure consistency and confidence in study data via standardized training and comprehensive oversight and documentation.
atom-international.org
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.
As a leader in rare diseases for over 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
alexion.com
Amicus Therapeutics is a leading, global biotechnology company with a clear and compelling mission: to develop and deliver transformative medicines for people living with rare diseases.
With extraordinary patient focus, Amicus strives to redefine expectations in rare disease.
For more information, visit us at amicusrx.com, and follow us on LinkedIn
.
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases.
Partnering with patients, healthcare professionals, and leading academic researchers, argenx aims to translate immunology breakthroughs into a world-class portfolio of accessible novel antibody-based medicines for patients living with chronic immune-mediated diseases.
argenx.com
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions.
Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.
biomarin.com
BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations.
Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market.
bridgebio.com
University of Rochester Center for Health and Technology (CHeT)
University of Rochester Center for Health and Technology (CHeT) Outcomes team develops and validates highly sensitive, disease-specific, regulatory-grade patient and caregiver reported outcome measures for therapeutic trials and drug labelling claims.
Our Health Indexes span 40+ neurological, neuromuscular, and movement disorders, have been used in over 100 clinical studies, and translated worldwide into over 40 languages.
URMC CHET – Outcomes
DEYMED Diagnostic is a leading Czech company with an established global existence, specialising in advanced medical diagnostic and treatment technologies for neurophysiology and psychiatry.
Founded in 1997, DEYMED has expanded its product range over the years to include EEG, EMG, TMS (Transcranial Magnetic Stimulation), PSG (Polysomnography), and BFB (Biofeedback) systems, trusted by healthcare providers worldwide for their precision, reliability, and ease of use.
deymed.com
Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.
We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease.
The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as a preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease.
At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities.
dyne-tx.com
Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions.
Guided by our approach to targeting the muscle as an organ, we have combined our foundational expertise in muscle biology and small molecule drug discovery to build our proprietary, muscle-focused platform. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases.
Edgewise Therapeutics
Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of intracellular therapeutics.
Entrada’s research and development includes several neuromuscular programs.
entradatx.com
The European Neuromuscular Centre (ENMC) was founded in 1992 by a group of European patient associations that dedicated itself to bring leading researchers and clinicians from all over the world together.
To achieve this goal, ENMC applies a concept unique in the scientific world, which consists of organising and financing workshops on application basis.
enmc.org/
Italfarmaco is a global pharmaceutical company operating in over 90 countries and committed to improving patients’ lives through pioneering solutions in women’s health, neurology, cardiovascular, and rare conditions.
Its rare disease unit is advancing therapies for Duchenne muscular dystrophy and amyotrophic lateral sclerosis, while also developing candidates for Becker muscular dystrophy and polycythemia vera. (EU-ITFRD-25-00006)
Italfarmaco – Rare Diseases
At Johnson & Johnson, we believe health is everything.
Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.
Learn more J&J Innovative Medicine EMEA
Follow us on
LinkedIn
LFB is a French biopharmaceutical group that develops, manufactures and markets plasma-derived medicinal products and recombinant proteins for the treatment of patients with serious and often rare diseases, in immunology, haemostasis, and intensive care.
Created in 1994, LFB today is a leading European company providing plasma-derived medicines to healthcare professionals.
groupe-lfb.com
Lupin Neurosciences, a specialty division of Lupin, is focusing on neuromuscular disorders following the launch of the company’s first Orphan Drug, NaMuscla® (mexiletine), licensed in Europe and UK for the treatment of non-dystrophic myotonia in adults.
The division’s aim is to develop treatment solutions within neuromuscular and movement disorder areas.
lupin-neurosciences.com
Merck has a long-standing legacy in neurology and immunology and aims to improve the lives of patients by addressing areas of unmet medical needs.
In addition to Merck’s commitment to MS, the company pipeline focuses on discovering potential new therapies in neuroinflammatory and immune-mediated diseases, including SLE, gMG, and NMOSD.
merckgroup.com
The MGBase Registry, supported by the MSBase Foundation, is a global collaborative registry for clinicians in Myasthenia Gravis (MG). It enables collection, tracking and evaluation of patient outcomes through free, secure, web-based tools accessible anywhere on any device, offering scientific support to advance research and improve understanding of MG worldwide.
mgbase.org
NADMED is a biotech company that offers NAD measuring technology. NADMED method is based on solid research at the University of Helsinki.
The method is the first to measure all four NADs quickly and efficiently with the accuracy of mass spectrometry. NADMED aims to be the standard in NAD measuring.
nadmed.com
Novartis Pharma AG
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease.
Our medicines reach nearly 300 million people worldwide.
novartis.com
Sanofi’s Specialty Care business focuses on neurology, rare diseases, rare blood disorders, oncology, and immunology. We help people with debilitating and complex conditions that are often difficult to diagnose and treat.
Our approach is shaped by our experience developing highly specialized treatments and forging close relationships with physician and patient communities. We are dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world.
Sanofi – Diseases & Conditions
Santhera Pharmaceutical is a Swiss commercial stage biopharmaceutical company committed to developing and commercializing innovative medicines for patients living with rare and other diseases with high unmet medical needs.
We are focusing on the development of treatments for neuromuscular diseases that currently lack treatment options, such as Duchenne muscular dystrophy (DMD).
santhera.com
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.
We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases.
sarepta.com
Satellos is a clinical-stage drug development company focused on restoring natural muscle repair and regeneration in degenerative muscle diseases. The company’s first-in-class small molecule drug, SAT-3247, targets AAK1 to replace dystrophin-associated signaling in muscle stem cells and is currently in clinical development as a potential disease-modifying treatment for DMD.
satellos.com
Scholar Rock is a late-stage biopharmaceutical company on a mission to discover, develop, and deliver life-changing therapies for serious diseases with high unmet need.
As a global leader in transforming growth factor beta (TGFβ) superfamily biology, the company is focused on advancing innovative treatments where protein growth factors are fundamental.
scholarrock.com
Takeda is focused on creating better health for people and a brighter future for the world.
We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines.
takeda.com
UCB Biopharma aims to improve lives by addressing disease challenges through innovative research in neurology, immunology, and more. Guided by patient perspectives, UCB delivers meaningful solutions globally, fostering collaboration, diversity, and care for people, communities, and the planet, from its Belgian headquarters to nearly 40 countries.
ucb.com
Vor Bio is a clinical-stage biotechnology company rapidly advancing telitacicept, designed to modulate the immune system and deliver meaningful treatment for people living with autoimmune disease.
By targeting the BAFF/APRIL pathway, Vor Bio aims to address disease at its source in serious B-cell driven conditions to improve patients’ daily lives.
vorbio.com
The World Federation of Neurology (WFN) is an association of neurological member societies representing 126 professional societies in all regions of the world. The mission of the WFN is to foster quality neurology and brain health worldwide, a goal we seek to achieve by promoting global neurological education and training.
wfneurology.org
