{"id":8538,"date":"2019-07-16T09:22:40","date_gmt":"2019-07-16T09:22:40","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=8538"},"modified":"2022-05-03T18:10:35","modified_gmt":"2022-05-03T18:10:35","slug":"exondys-51-slows-down-respiratory-decline","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/exondys-51-slows-down-respiratory-decline\/","title":{"rendered":"Exondys 51 slows down respiratory decline"},"content":{"rendered":"

Thanks to<\/span> Bio Space<\/a>, IOS Press<\/a>, Science Daily<\/a> for\u00a0the content of this blog.<\/span><\/span><\/p>\n

Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary function becomes progressively impaired as the dystrophic process affects respiratory muscles, including the diaphragm. Strategies to arrest this severe gradual deterioration are needed to extend lives and improve quality of life.<\/strong><\/p>\n

Results of three clinical trials using eteplirsen show promising results<\/h4>\n

Sarepta, along with Harvard Medical School, The Children\u2019s Hospital of Philadelphia, Nationwide Children\u2019s Hospital, the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center and Ohio State University\u00a0published results<\/a>\u00a0<\/span>from three trials in the\u00a0Journal of Neuromuscular Diseases<\/em>.<\/p>\n

This study conducted in three clinical trials supports that\u00a0Sarepta Therapeutics<\/span><\/a>\u2019 Exondys 51 (eteplirsen) slows respiratory decline in Duchenne muscular dystrophy (DMD).<\/p>\n

The respiratory decline in patients treated with eteplirsen was significantly lower, and this was true across all stages of the disease evaluated.<\/p>\n

As the disease progresses, patients require increasing levels of clinical treatment. Patients are at increased risk of death once this respiratory decline reaches a critical threshold.<\/p>\n

Eteplirsen may slow the rate of respiratory decline and therefore may delay time to milestones of decrease. This may have notable positive implications on quality of life. Longer-term follow-up is needed.<\/strong><\/p>\n

Pulmonary function<\/h4>\n

The pulmonary function can be measured by assessing different parameters of lung function. As an example, the total amount of air that can be moved through the lungs after a maximal inspiration and then exhalation (forced vital capacity [FVC]). The FVC measures output of inspiratory and expiratory muscles. This is an excellent measure of respiratory function reserve and is widely used in DMD to assess respiratory function.<\/p>\n

About Exondys 51<\/h4>\n