Living with Charcot-Marie-Tooth disease (#CMT) means navigating a growing list of physical challenges that can make even everyday tasks difficult. As the disease progressively damages the peripheral nerves, patients experience a combination of #muscle weakness, impaired balance, poor dexterity, chronic pain, and declining muscle control that tends to worsen over time. Symptoms can vary considerably depending on the subtype, but muscle weakness in the upper and lower limbs remains a core feature across the CMT spectrum. Many individuals experience hand weakness and dexterity problems, which are difficult to correct with supportive braces, in addition to difficulties fine motor tasks, such as buttoning a shirt, as well as foot drop, balance problems, and a gradual loss of independence as the disease progresses. Despite the significant burden these symptoms place on patients and caregivers, there are currently no approved drug treatments that address them directly. Management remains primarily supportive, relying on orthotics, physical or occupational therapy, and intensive surgical intervention to help address mobility challenges, rather than treating the underlying cause of weakness and disease. #CharcotMarieToothDisease #RareDisease #NeuromuscularDisease #PatientVoice #CMTAwarenessMonth #Innovation

Less than 3 weeks to go! 🏃 On November 1, Maja Nielsen, Michael Voldsgaard Clausen, and Hang Nguyen Nielsen will take on 26.2 miles through all five boroughs of New York City as part of the #NMD Pharma team at the TCS New York City Marathon. The team will be raising money in support of the @CMTNeuropathy Foundation to help the more than three million people living with Charcot-Marie-Tooth disease (#CMT). CMT causes progressive muscle weakness and loss of balance, making everyday movement a challenge. Each mile run is a chance to raise awareness and fund the research, education, and #advocacy that can change lives within the community. Want to be part of the journey? Every donation, big or small, helps bring better treatments within reach. Donate here: fundraise.givesmart.com/vf/C… #CharcotMarieTooth #TCSNYCMarathon #NYCMarathon2026 #NeuromuscularDisease #PassionForMovement #StrongerTogether #RareDisease

𝗕𝗲𝗵𝗶𝗻𝗱 𝗲𝘃𝗲𝗿𝘆 𝗱𝗮𝘁𝗮 𝗽𝗼𝗶𝗻𝘁 𝗶𝘀 𝗮 𝗽𝗲𝗿𝘀𝗼𝗻 Last month, we welcomed Reagan Warren, a 17-year-old patient advocate living with Charcot-Marie-Tooth disease (CMT), to NMD Pharma to take part in a summer “Capstone” project (similar to a short summer internship). The project was designed to help Reagan gain insight into biopharmaceutical companies and how they innovate, collaborate and communicate with others to develop drug therapies to help those with rare diseases. CMT is a rare neuromuscular disease and is a serious and progressive nerve disorder that affects the body’s muscles, impacting daily activities, qualify of life and ability to be independent *. During her visit, Reagan attended the Peripheral Nerve Society (PNS) Annual Meeting in Maastricht alongside members of our team, visited our headquarter offices and laboratories in Aarhus, met colleagues across many functions within the company, and gained first-hand insight into how research is translated into potential new therapies. The learning went both ways. Reagan shared her personal experience of living with CMT, offering valuable insight into the realities behind the science - from multiple surgeries and rehabilitation to the impact the disease has had on her independence, education, and family life. She also spoke about the importance of continued research and innovation for people living with CMT. As Reagan told us: "𝘐𝘵 𝘮𝘦𝘢𝘯𝘴 𝘦𝘷𝘦𝘳𝘺𝘵𝘩𝘪𝘯𝘨 𝘵𝘰 𝘶𝘴 𝘢𝘴 𝘱𝘢𝘵𝘪𝘦𝘯𝘵𝘴 𝘵𝘩𝘢𝘵 𝘺𝘰𝘶 𝘨𝘶𝘺𝘴 𝘢𝘳𝘦 𝘥𝘰𝘪𝘯𝘨 𝘸𝘩𝘢𝘵 𝘺𝘰𝘶 𝘢𝘳𝘦 𝘥𝘰𝘪𝘯𝘨." Her visit was a timely reminder that behind every experiment, every data point, and every clinical milestone is a person living with the disease we are working to treat. Our collaboration with Reagan continues beyond this visit. Later this summer, Reagan will join members of NMD's leadership team in meetings with potential investors and life science bankers to understand the process and importance of continued financial investment in companies on the way to developing new medicines. She will also gain insight and prove input on the different ways that companies like NMD Pharma communicate with physicians, investors, patients and families and hopefully provide some insight and guidance for ways we can communicate more effectively! Thank you, Reagan, and the Warren family, for partnering with us and for helping us better understand the needs and perspectives of people living with CMT. *CMT is a serious and progressive nerve disorder that affects the body’s muscles. From early signs like frequent tripping, toe-walking, and trouble with fine motor skills, those living with CMT may experience symptoms such as: · Hand/arm weakness (reduced grip strength) · Difficulty with mobility (walking, stair climbing) · Balance · Ankle weakness (foot drop) · Muscle fatigue · Difficulty breathing #RareDisease #PatientVoice #Biotech #DrugDevelopment #Innovation

𝗔 𝗻𝗲𝘄𝗹𝘆 𝗶𝗱𝗲𝗻𝘁𝗶𝗳𝗶𝗲𝗱 𝗺𝗲𝗰𝗵𝗮𝗻𝗶𝘀𝗺 𝗼𝗳 𝗮𝗴𝗲-𝗿𝗲𝗹𝗮𝘁𝗲𝗱 𝗺𝘂𝘀𝗰𝗹𝗲 𝘄𝗲𝗮𝗸𝗻𝗲𝘀𝘀 𝗺𝗮𝘆 𝗼𝗽𝗲𝗻 𝗮 𝗱𝗶𝗳𝗳𝗲𝗿𝗲𝗻𝘁𝗶𝗮𝘁𝗲𝗱 𝘁𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰 𝗽𝗮𝘁𝗵 𝗶𝗻 𝘀𝗮𝗿𝗰𝗼𝗽𝗲𝗻𝗶𝗮—𝗮𝗻𝗱 𝗯𝗿𝗼𝗮𝗱𝗲𝗻 𝘁𝗵𝗲 𝗽𝗼𝘁𝗲𝗻𝘁𝗶𝗮𝗹 𝗼𝗳 𝗡𝗠𝗗 𝗣𝗵𝗮𝗿𝗺𝗮’𝘀 𝗖𝗹𝗖-𝟭 𝗽𝗹𝗮𝘁𝗳𝗼𝗿𝗺 𝗯𝗲𝘆𝗼𝗻𝗱 𝗿𝗮𝗿𝗲 𝗻𝗲𝘂𝗿𝗼𝗺𝘂𝘀𝗰𝘂𝗹𝗮𝗿 𝗱𝗶𝘀𝗲𝗮𝘀𝗲𝘀. Today, we announced the publication of new clinical and preclinical research in @jclinicalinvest showing that older adults with clinically significant muscle weakness — a hallmark of sarcopenia — exhibit neuromuscular junction transmission failure, with greater transmission impairment associated with greater muscle weakness. Importantly, inhibition of the skeletal muscle-specific ClC-1 ion channel enhanced muscle excitability, improved neuromuscular transmission, and increased muscle function in aged rodent models exhibiting deficits similar to those observed in weak older adults. These findings provide a biologically grounded rationale for targeting ClC-1 in moderate-to-severe sarcopenia, a large and underserved patient population with no approved pharmacologic therapies. The publication also supports NMD Pharma’s broader strategy of building a multi-asset ClC-1 portfolio designed to restore reliable skeletal muscle activation across both rare neuromuscular diseases and larger, more prevalent skeletal muscle disorders. Building on the positive results from the Phase 2 SYNAPSE-CMT study, NMD Pharma is advancing ignaseclant toward clinical studies designed to support registration in Charcot-Marie-Tooth disease (CMT), while next-generation ClC-1 programs are intended to expand the platform into moderate-to-severe sarcopenia and other areas of significant unmet need. Congratulations to the group of #researchers, including our CEO, Thomas Holm Pedersen, and Dr. W. David Arnold, for publishing these important findings! Read the full press release: nmdpharma.com/news/late-brea… Read the publication in JCI: jci.org/articles/view/190646… #NMDPharma #Sarcopenia #MuscleHealth #NeuromuscularDisease #Biotechnology #DrugDiscovery

This November, several #NMD Pharma team members will be running the TCS New York City Marathon in support of the Charcot-Marie-Tooth disease (#CMT) community! We’re proud to once again be partnering with @CMTNeuropathy (HNF) to raise awareness and funding for critical research on CMT, a progressive neuromuscular disorder that impacts over three million people worldwide. On November 1, Maja Nielsen, Michael Voldsgaard Clausen, and Hang Nguyen Nielsen will put their passion for movement into action by running in support of those who face difficulties with muscle strength and function. Every mile is a reminder that progress, like the search for better treatments, is built one step at a time. We’re proud to collaborate with organizations like the HNF that are making a real difference in the lives of people living with CMT through #advocacy, education, and research investment. Donate to the cause here: fundraise.givesmart.com/vf/C… #CharcotMarieTooth #TCSNYCMarathon #NeuromuscularDisease #PassionForMovement #StrongerTogether #RareDisease