Thelansis

  Capricor Therapeutics, a biotechnology company dedicated to developing transformative cell and exosome-based therapeutics for rare diseases, has announced the initiation of its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA). This application seeks full approval for their therapy, deramiocel, aimed at treating patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. This significant step highlights Capricor Therapeutics’ commitment to addressing the unmet medical needs of those affected by DMD cardiomyopathy. Publish Date: 09-10-2024   Source: Capricor Therapeutics Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by the progressive weakening and wasting of muscles, affecting skeletal, smooth, and cardiac muscle tissues. The underlying cause of muscle damage is the absence of the sarcolemmal protein dystrophin due to variations in the DMD gene located on Xp2...

 Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by the progressive weakening and wasting of muscles, affecting skeletal, smooth, and cardiac muscle tissues. The underlying cause of muscle damage is the absence of the sarcolemmal protein dystrophin due to variations in the DMD gene located on Xp21.2. Symptoms of DMD typically manifest in early childhood, with affected boys often experiencing delayed walking, typically after 18 months, along with speech and developmental delays. Individuals with DMD may also face autism and behavioral issues, including ADHD, anxiety, and obsessive-compulsive disorder. Unfortunately, untreated children with DMD rarely achieve the ability to run or jump. The condition progresses rapidly, leading to a characteristic waddling gait and a positive Gowers' sign. Climbing stairs becomes challenging, and frequent falls occur. Independent walking is typically lost between the ages of 6 and 13, with an average age of 9.5 in non-stero...

  Duchenne muscular dystrophy (DMD) is caused by a lack of dystrophin, a protein that aids in the maintenance of muscle cells. Symptoms usually appear in early childhood, between the ages of 3 and 5. The disease primarily affects boys but can also affect girls in rare cases. Because the gene that can carry a DMD-causing mutation is on the X chromosome, DMD is inherited in an X-linked pattern. Mutations in the DMD gene cause progressive muscle weakness, loss of independent ambulation by early adolescence, and premature death from cardiorespiratory complications. The global DMD prevalence was 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population, while the global DMD birth prevalence was 19.8 per 100,000 live male births. Thelansis’s “Duchenne Muscular Dystrophy (DMD) Market Outlook, Epidemiology, Competitive Landscape , and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription shar...