Charcot-Marie-Tooth (CMT) disease is a rare, chronic and sensory neuropathy that causes weakness of the limbs and progresses gradually over time. It can lead to atrophy, sensory loss and complete limb dysfunction. However, rare, the condition is the most common inherited disorder that damages peripheral nerves with an estimated global prevalence of  2.6 million people worldwide (NINDS). As per DelveInsight estimates, the total Charcot-Marie-Tooth prevalence in the 7MM (the US, EU5 and Japan) was 263,835 in 2020 that is expected to surge in the coming years. Moreover, the estimates suggest that females are prone to get diagnosed with CMTdisease as compared to males. 

CMT disease hampers day-to-day routine, affecting the professional and personal lives of patients. However, the rarity of the condition arises several unique challenges that widen the treatment gap. For instance, there is a lack of statistical epidemiological data that could help jot down the global approximate burden of CMT disease. There is a serious lack of patient-reported outcomes that helps in providing deep insight into the disease pathophysiology, thereby facilitating better disease understanding. 

The condition has no cure at hand. Further, there exist no curative therapies in the Charcot-Marie-Tooth disease market. The present CMT disease therapeutics market is purely based on supportive and symptomatic management that includes occupational therapies, physiotherapy, ankle-foot orthoses (AFO), and shoe inserts. The care involves occupational therapy followed by physiotherapy, with approximately 30% of the total CMT disease diagnosed population resorting to foot surgeries. To address the neuropathic pain, muscle pain or pain in the joints, the Charcot-Marie-Tooth therapy market comprises painkillers such as tricyclic antidepressants (TCAs) or anticonvulsant medication. 

Even so, early treatment helps in better treatment of the condition and early beginning of physical therapy such as muscle strength training, muscle and ligament stretching, and moderate aerobic exercise to maintain mobility, flexibility and strength deliver significant advantages. However, many times a patient does not get diagnosed until in the late sixties and by that time, the symptoms get severe. Besides, the cost associated with the diagnosis is also one of the significant hurdles that delay the timely diagnosis of the condition. 

Thus, missed or late diagnosis is a serious concern; however, it can be due to the heterogeneity of the condition, late age of onset of symptoms, slow and gradual development of symptoms, and so on. Additionally, a lack of curative and approved therapies in the CMT disease market is a serious unmet need. 

However, a severe dearth of effective and safe therapies in the Charcot-Marie-Tooth therapeutic market has managed to pull in several pharma and biotech companies exploring and developing many novel therapies. Further, the CMT disease market has been witnessing tremendous growth in the R&D, healthcare expenditure and overall demand for better therapies and treatment that is going to boost the market growth exponentially. DelveInsight estimates that the Charcot-Marie-Tooth therapy market is anticipated to surge at a CAGR of 25.42% for the Study Period 2018–2030, with the USA accounting for the majority of the market share in the 7MM. Key pharmaceutical companies such as Pharnext, MedDay Pharmaceuticals, among others are proactively working in the domain; however, the CMT disease pipeline constitutes only a few therapeutic agents such as MD1003 and PXT3003 in advanced stages of clinical trials. The scenario depicts immense untapped opportunities in the space for pharma and biotech companies to tap and lead in the CMT disease market domain. 
However, there is still a requirement for a streamlined process to ensure proper stratification of patients during clinical studies, better biomarkers to facilitate accurate diagnosis and effective treatment protocols. Nevertheless, the Charcot-Marie-Tooth disease market size is expected to experience a surge owing to the entry of upcoming novel CMT disease pipeline therapies, increasing prevalence of the disease along with an improved scenario of genetic sequencing techniques, identification of mutant genes and proteins responsible for the condition, a better understanding of the disease pathophysiology and a promising outcome of the research taking the path towards the cure.