The UK Oculopharyngeal Muscular Dystrophy (OPMD) 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.
Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, adult-onset, progressive myopathy characterized by progressive eyelid ptosis, ophthalmoplegia, dysphagia, dysarthria, and proximal limb weakness. OPMD is a genetic muscle disorder that mainly affects the muscle of the upper eyelids and throat. OPMD is caused by an expansion in the polyalanine tract in the PABPN1 gene (14q11.2), which leads to overexpression of a mutant protein, polyadenylate-binding protein 2, and consequently to the accumulation of nuclear aggregates in the muscles.
Disease onset occurs in the fifth to sixth decade of life. Early signs include ptosis, ophthalmoplegia without diplopia, limb weakness, dysarthria, and dysphagia. Symptoms usually begin after the age of 45 years, and ptosis is the most common presenting feature. In several cases, limb weakness has preceded dysphagia. In 5-10% of patients, the disease is more severe, with ptosis and dysphagia presenting before the age of 45 and incapacitating distal leg weakness occurring before the age of 60. The manifestations of autosomal recessive OPMD usually present later (after the age of 60) than those of the autosomal dominant form.
OPMD is a rare disorder that affects males and females in equal numbers. The OPMD is among the 30 inherited diseases declared by WHO and its results in the weakening and worsening of muscles. OPMD affects less than 5 in 10,000 people in the EU. According to a study, in Europe, the estimated prevalence of oculopharyngeal muscular dystrophy is 1 in 100,000 people. The analysis demonstrates the phenotypic and genotypic characteristics of 31 patients with OPMD in the UK. Ptosis was the first reported symptom in two-thirds of the patients, and half of the subjects studied had evidence of ophthalmoplegia. The PABP2 analysis is a reliable non-invasive diagnostic test for OPMD in the UK population.
Market Growth Drivers
The major driving factor involved in the OPMD therapeutics market is the increased incidence and prevalence of oculopharyngeal muscular dystrophy in the middle-aged population. Conventional therapy mainly focused on symptomatic treatment of Oculopharyngeal Muscular Dystrophy and having lots of off-target effects, this factor providing the additional opportunity for key players present in the OPMD treatment market to expand their research and development activities and expected to drive the growth of oculopharyngeal muscular dystrophy treatment market during the forecast period.
Additionally, the introduction of new disease-modifying and novel drug therapies, increased drug development activities are expected to drive the oculopharyngeal muscular dystrophy treatment market. Furthermore, gene therapies such as mutation suppression, exon skipping are also anticipated to push the OPMD treatment market during the forecast period.
On the contrary, the high cost of the novel therapies and lack of awareness among individuals are some factors that are expected to hamper the growth of the oculopharyngeal muscular dystrophy treatment market during the forecast period.