The Australian X-linked Myotubular Myopathy (XLMTM) Therapeutics Market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
X-linked myotubular myopathy (XLMTM) is a rare genetic neuromuscular disorder that is characterized by muscle weakness that is most typically severe but can range from mild to profound. Symptoms are often present at birth, though may develop later in infancy or early childhood. Rarely, symptoms may not present until adolescence or adulthood. Common symptoms include mild to profound muscle weakness, diminished muscle tone (hypotonia or “floppiness”), feeding difficulties, and potentially severe breathing complications (respiratory distress). Feeding difficulties and respiratory distress develop because of weakness of the muscles that are involved in swallowing and breathing. XLMTM is caused by mutations to the myotubularin (MTM1) gene. The disorder is inherited as an X-linked condition. The disorder predominantly affects males, but female carriers, while typically asymptomatic, can develop a range of symptoms.
XLMTM primarily affects males. Some carrier females may develop mild symptoms associated with the disorder. The exact incidence of the disorder is unknown, but estimated incidence of 17 per million births for severe X-linked myotubular myopathy (XLMTM), the main subtype of CNM, corresponds to an estimated prevalence of 72 in Australia.
Rise in incidence of X-Linked Myotubular Myopathy (XLMTM) is likely propel the market. Moreover, increase in research & development by key players is anticipated to drive the market. Several players are investing in development of products/therapies like Gene therapy that is being studied as an approach to therapy for XLMTM. Furthermore, introduction of new products by key players is anticipated to drive the X-Linked Myotubular Myopathy (XLMTM) market. Emergence of new therapies for acute phase or prevention of X-Linked Myotubular Myopathy (XLMTM) creates lucrative opportunity for the market.
However, rising treatment costs and a lack of awareness among patients and physicians about X-Linked Myotubular Myopathy are among the major factors acting as restraints, and will continue to pose a challenge to the X-Linked Myotubular Myopathy Therapeutics market in the forecast period.