Aug 23, 2024
Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the...
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Aug 20, 2024
Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treati...
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Aug 20, 2024
Duchenne muscular dystrophy (DMD) is a rare genetic disorder usually diagnosed in young boys, gradually weakening muscles across the body until the heart or lungs fail. Symptoms often show up by the age of 5; as the disease progresses, patients tend to lose the ability to walk around the age of 12. As per a recent ...
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Aug 20, 2024
A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. In 2023, the total number of prevalent cases of Duchenne Muscular Dystrophy in the 7MM (The US, EU5, and Japan) was around 31,400. The United States encompasses the highest prevalent population of Duche...
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Jun 19, 2024
Duchenne muscular dystrophy (DMD) is a severe genetic disorder primarily affecting young boys between the ages of two and three. As per Delveinsight’s estimates, there were approximately 16K DMD patients in 2023 in the United States, with ˜75% of the patients below the age of 15. This condition leads to progressive...
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Jun 30, 2023
Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...
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Roche’s HER2-Positive Breast Cancer Treatment Franchise
Aug 13, 2024
Transforming Multiple Myeloma Treatment: The Promise of Novel Drug Classes
Jul 24, 2024
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