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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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How Pharma Companies Can – And Should – Improve Social Determinants of Health

Intouch Solutions

SDOH can drive as much as 80% of health outcomes 3,4 A person’s zip code, not their genetic code, is the stronger predictor of their health! Pharmaceutical companies can sponsor as well as provide these leaders. Pharmaceutical companies can increase diversity in their clinical trials. 1 Why are SDOH important?

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How using AI in clinical trials accelerates drug development

Pharmaceutical Technology

But before pharmaceutical companies can go to market with a breakthrough drug, they need to ensure safety and efficacy through clinical trials. Pharma R&D teams are solving this problem by leveraging the power of artificial intelligence (AI) in clinical trials to save time and money.

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Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies

Delveinsight

Key pharmaceutical companies such as Pharnext, MedDay Pharmaceuticals, among others are proactively working in the domain; however, the CMT disease pipeline constitutes only a few therapeutic agents such as MD1003 and PXT3003 in advanced stages of clinical trials.

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Is a lack of real-time data holding trials back?

pharmaphorum

Yet, for clinical trials worldwide, real-time access to data is not the norm – and that lack can create a cascading set of issues affecting every aspect of a study. What does this mean for clinical trials? Analysts at Pharma Intelligence decided to find out, surveying professionals involved in clinical data management.

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GSK turns to AI specialist Tempus for R&D productivity boost

pharmaphorum

GSK is the latest pharma group to tap into that resource, with the aim of teasing out new drug targets, improving the design of its clinical trials, and accelerating recruitment of test subjects. The two companies have been working together for some time.

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Sohonos (Palovarotene) Sets Milestone as First Drug for Ultra-Rare Bone Disease

XTalks

XTALKS WEBINAR: The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life Live and On-Demand: Thursday, September 28, 2023, at 11am EDT (4pm BST/UK) Register for this free webinar to learn about the key considerations when operationalizing pediatric rare disease studies with decentralized elements.

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