Alexion Pharma GmbH | Booth #208

Alexion, AstraZeneca Rare Disease, is a leader in rare diseases for 30 years focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. (alexion.com) 

Alnylam Pharmaceuticals | Booth #113

Founded in 2002, Alnylam has led the translation of RNA interference (RNAi) from Nobel Prize-winning discovery into an innovative, new class of medicines. Alnylam’s vision is to harness the potential of RNAi therapeutics to transform the lives of people living with diseases for which treatment options are limited or inadequate. (alnylam.be)

Amylyx Pharmaceuticals | Booth #312

Amylyx Pharmaceuticals is a clinical-stage biopharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. (amylyx.com)

Astellas Gene Therapies | Booth #209

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. We are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and will also advance additional Astellas gene therapy programs toward clinical investigation. (astellasgenetherapies.com) 

Dyne Therapeutics | Booth #204 

Dyne Therapeutics is building a leading muscle disease company dedicated to advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue seen with other approaches. (dyne-tx.com) 

European Neuromuscular Centre (ENMC)

The mission of the European Neuromuscular Centre (ENMC) is to encourage and facilitate communication and collaboration in the field of neuromuscular research with the aim of improving diagnosis and prognosis, finding effective treatments and optimizing standards of care to improve the quality of life of people affected by neuromuscular disorders. (enmc.org)

Grupo Ferrer | Booth #109

At Ferrer we want to make a positive impact in society and we do so by reinvesting a significant part of our profits in initiatives with social and environmental impact, as well as in our people. In order to fulfill our purpose, we offer transformative therapeutic solutions, with an increasing focus on pulmonary vascular and interstitial lung diseases and neurological disorders. (ferrer.com)

iThera Medical | Booth #305

iThera Medical develops and markets optoacoustic imaging (OAI) systems for preclinical and clinical research. OAI utilizes the photoacoustic effect to visualize and quantify optical contrast in deep tissue, at high spatiotemporal resolution. In the area of neuromuscular diseases, OAI has shown the potential to assess changes in tissue composition related to inflammation and fibrosis. (ithera-medical.com)

Janssen Research and Development | Booth #103

At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson&Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. Learn more at janssen.com.  

Journal of Neuromuscular Diseases | Table 310

The Journal of Neuromuscular Diseases facilitates progress in understanding the molecular genetics, pathogenesis, diagnosis, and treatment of neuromuscular diseases. Guided by Editors-in-Chief Carsten G. Bönnemann and Hanns Lochmüller, and published by IOS Press, JND is a vehicle for research that will improve understanding and lead to effective treatments of neuromuscular diseases. (iospress.com)

Lupin Neurosciences | Booth #211

Lupin Neurosciences, a division of Lupin Atlantis Holdings SA, is focusing on neuromuscular disorders following the launch of the company’s first Orphan Drug, NaMuscla® (mexiletine), indicated for the treatment of myotonia symptoms in adult patients with non-dystrophic myotonia. NaMuscla® is currently commercially available in Germany, UK, France, Spain and Norway. Please consult summary of product characteristics, available here EU-LUP-2201-00005. (lupin.com)

Novartis Gene Therapies | Booth # 100

Novartis Gene Therapies is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments. We are powered by an extensive manufacturing footprint enabling us to bring gene therapy to patients around the world. (novartis.com)

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PTC – Duchenne Muscular Dystrophy (DMD) | Booth #105

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. To learn more about PTC, please visit us at ptcbio.com and follow us on Facebook, LinkedIn, Twitter and Instagram.

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Roche | Booth #309

Neuroscience is a cornerstone of our future. We will continue to push the boundaries of scientific understanding, together with our partners, to achieve clinical advancements and solve some of the greatest challenges in neuroscience today. Our hope is to create a tomorrow where nervous system disorders no longer limit human potential – to preserve what makes us who we are. (roche.com)

Sanofi | Booth #301

Sanofi is an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our teams across the world strive to transform the practice of medicine, turning the impossible into the possible for patients. (sanofi.com) 

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Sarepta Therapeutics | Booth #111

At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. (sarepta.com)

Scholar Rock, Inc. | Booth #107

Scholar Rock is focused on the discovery and development of innovative medicines for treatment of serious diseases. Following positive results from TOPAZ, apitegromab, a selective inhibitor of active myostatin in skeletal muscle, is advancing through development with SAPPHIRE, a double-blind, placebo-controlled, phase 3 RCT, in patients with later onset SMA. (scholarrock.com)

SERB Specialty Pharmaceuticals | Booth #206

SERB Specialty Pharmaceuticals is bringing lifesaving drugs that meet high unmet medical needs. SERB has extended its Rare Diseases portfolio in both Neurology (with treatments for severe epileptic and Lambert Eaton Myasthenic Syndrome) and Metabolic Disorders. SERB makes available the only EMA registered drug for symptomatic treatment of adult LEMS patients. (serb.eu)

Takeda International AG| Booth #114

Takeda is a patient-focused, R&D-driven biopharmaceutical company committed to bringing better health to people worldwide since 1781. Takeda aspires to unlock the potential of plasma in our relentless drive to improve the lives of patients with chronic inflammatory demyelinating polyneuropathy, multifocal motor neuropathy, and other rare auto immune mediated diseases. (takeda.com)

UCB | Booth #108

UCB, Brussels, Belgium is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system.  Visit us on LinkedIn, Twitter, Facebook or Instagram. (ucb.com)

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