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Highly anticipated as new disease-modifying treatments for Duchenne muscular dystrophy (DMD), therapeutics by BioMarin Pharmaceutical (Kyndrisa; drisapersen) and Sarepta Therapeutics (eteplirsen; AVAVI-4658) both recently received negative FDA...
Substitutions, deletions and duplications in the dystrophin gene lead to either the severe Duchenne muscular dystrophy (DMD) or mild Becker muscular dystrophy depending on whether out-of-frame or in-frame transcripts are produced. We identified a DMD case (GSΔ44) where the correlation between genotype and phenotype is not respected, even if carrying a typical Duchenne mutation (exon 44 deletion) a Becker-like phenotype was observed. Here we report that in this patient, partial restoration of an in-frame transcript occurs by natural skipping of exon 45 and that this is due to the lack of Celf2a, a splicing factor that interacts with exon 45 in the dystrophin pre-mRNA. Several experiments are presented that demonstrate the central role of Celf2a in controlling exon 45 splicing; our data point to this factor as a potential target for the improvement of those DMD therapeutic treatments, which requires exon 45 skipping.
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, today announced that the European Commission (EC) has granted orphan medicinal product designation to CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD). CAT-1004 is designed to inhibit activated NF-kB, a protein that plays an important role in muscle health. CAT-1004 is a potential disease-modifying therapy being developed to reduce muscle inflammation and degeneration and promote muscle regeneration in patients affected by DMD regardless of the underlying dystrophin mutation. The U.S. Food and Drug Administration (FDA) has previously granted Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for CAT-1004 for the treatment of DMD.
Apple is expanding its ResearchKit program with three new studies and three new partner universities. This time, Apple will help research studies on autism, epilepsy and melanoma.As a reminder, ResearchKit is a set of tools for collecting participant consent, conducting surveys and assigning active tasks to participants that they can perform to help researchers achieve specific study goals. It lets researchers leverage Apple devices for their studies.If users agree to share these sets of data, researchers can collect data from iPhones, Apple Watches and now iPads as well as iOS accessories, which is great if you want to measure blood pressure, glucose levels and more. Researchers can take advantage of the accelerometers, gyroscope, heart monitor, GPS sensor, microphone and more.
Genetic ablation of P2RX7 can improve muscle function and partially correct cognitive impairment and bone loss in a mouse model of Duchenne muscular dystrophy, according to a study published this week in PLOS Medicine. The study, conducted by Dariusz Gorecki of the School of Pharmacy and Biomedical Sciences, University of Portsmouth, UK and colleagues, additionally suggests that P2RX7 antagonists can reduce certain DMD phenotypes in these mice.
In the not-so-distant past, patients had only one source for healthcare expertise — their personal physicians. They relied on doctors to monitor their symptoms, track changes in their health, manage their diseases and personalize their care. But in recent years, mobile technology and the Internet of Things (IoT) has changed all that.Now, armed with wearables and mobile healthcare apps, individuals can monitor their own health data and get continuous guidance and information from the devices they carry with them every day.But the availability of digital health tools is a double-edged sword for the medical industry. Patients are becoming more engaged in their own wellness and taking greater responsibility for staying healthy, which leads to better outcomes. On the other hand, as they rely more on these tools, are they relying less on the robust insights and expertise of medical professionals?
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