NMD Pharma to Present Results from the Charcot-Marie-Tooth ESTABLISH Study at the Peripheral Nerve Society Annual Meeting
Data provide valuable insights into the role of neuromuscular junction failure as a disease characteristic
Aarhus, Denmark, 9 June 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for neuromuscular disorders, today announces that it will present results from the ESTABLISH1 Study at the Peripheral Nerve Society Annual Meeting (PNS) being held from 17-20 June 2023 in Copenhagen, Denmark.
The ESTABLISH study is an international observational clinical study in collaboration with Aarhus University and The Ohio State University to assess neuromuscular function in patients with Charcot-Marie-Tooth disease types 1 and 2, an inherited neuromuscular disorder of the peripheral nerves characterized by weakness and fatigue. The aim of this study was to understand the role of neuromuscular junction (NMJ) failure as a new disease characteristic in CMT types 1 and 2, as suggested by preclinical studies and evidence from other neuromuscular disorders.
Full details of the poster presentation are:
Title: Neuromuscular Junction Transmission Dysfunction in Patients with Charcot-Marie-Tooth Disease – The ESTABLISH Study
Authors: Thomas Skjærlund Grønnebæk, Helga Haahr-Lillevang, Kristina Kelly, Amy Bartlett, Hatice Tankisi, Jane Bold, John Hutchison, Jorge Quiroz, Thomas Holm Pedersen, Henning Andersen2, William David Arnold2
Date and Time: 19 June 2023, 18:00-20:00 CEST
To view the full abstract, please follow the following link: https://pnsociety.com/2023-annual-meeting/
1Exploring SynapTic ABnormaLitIeS in Hereditary neuropathies
2Co-Principal Investigators
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Contacts
NMD Pharma A/S
Thomas Holm Pedersen, CEO
E-mail: contact@nmdpharma.com
Consilium Strategic Communications
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: NMDPharma@consilium-comms.com
Tel: +44 (0)20 3709 5700
About NMD Pharma
NMD Pharma A/S, is a private biotech company leading in the development of novel first-in-class therapies for severe neuromuscular disorders. The Company was incorporated as a spin-off from Aarhus University, Denmark in 2015 and was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. NMD Pharma received initial seed financing in 2016 and have since raised ~€80 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital.
About the Department of Clinical Medicine: Aarhus, Denmark
Department of Clinical Medicine is Denmark’s largest health science institute conducting research in almost all medical specialities and hosting a large number of research centres. Most of our staff are employed part-time at the department and part-time as clinical staff at Aarhus University Hospital or one of the four regional hospitals in Central Denmark Region. The close collaboration between Aarhus University and Aarhus University Hospital ensures fast implementation of research results in clinical practice to the benefit of patients. Department of Clinical Medicine is located at Aarhus University Hospital, which has been awarded Denmark’s best hospital 13 times in a row.
About The Ohio State University
The Ohio State University Wexner Medical Center, based in Columbus, Ohio, USA, is a proud part of one of America’s largest and most comprehensive universities. On the university’s main Columbus campus, more than 56,000 students are able to choose from 14 colleges, 175 undergraduate majors and 240 master’s, doctoral and professional degree programs. Ohio State is consistently ranked as Ohio’s best and one of the nation’s top-20 public universities.
The university’s research innovations have attained world-class status, particularly in critical areas such as global climate change, cancer, infectious disease, neurosciences, advanced materials and ag-bio products that feed and fuel the world.
About Charcot-Marie-Tooth (CMT) disease
CMT encompasses a group of hereditary sensory and motor neuropathies that cause damage to peripheral nerves. Damage caused by CMT worsens over time and can result in the loss or alteration of sensation and atrophy of muscles in the legs, feet, arms, and hands. There are several types of CMT that are differentiated by their effects on neurons and inheritance patterns caused by mutations on the X chromosome - both result in either abnormalities in myelination, axonal degeneration, or both. CMT affects approximately 2.6 million individuals worldwide with symptoms appearing typically in adolescence or early adulthood, however, onset of the disease can occur at any age.