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Three drugs secure ‘rare paediatric disease designation’ in the US

Drug Discovery World

Three drugs have been granted ‘rare paediatric disease designation’ (RPDD) in the US by the Food and Drug Administration (FDA). The FDA grants RPDD for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and primarily affect patients less than 18 years of age.

Disease 147
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AI identifies drug candidates for respiratory syncytial virus

Drug Discovery World

Potential new drug candidates have been identified by a respiratory syncytial virus (RSV) artificial intelligence programme, as health officials warn of a “tripledemic” of SARS-CoV-2, influenza and RSV. . The post AI identifies drug candidates for respiratory syncytial virus appeared first on Drug Discovery World (DDW).

Virus 130
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Research reveals therapeutic potential of mRNA in rare diseases

Drug Discovery World

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

Disease 147
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Meet the Researcher: Liam Tremble

Drug Discovery World

Where do you work, and can you tell us what you’re working on at the moment?  I work at Poolbeg Pharma, where we are developing novel treatments for diseases with unmet medical needs and large addressable markets, with programmes in oncology, infectious diseases and endocrinology.

Research 221
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Covid researchers launch Monkeypox study  

Drug Discovery World

The trial will see participants receive either a 14-day course of 600 mg tecovirimat twice daily or a matched placebo treatment. As a community-based trial, participants will take the treatment or placebo in their own homes. The trial looks to include at least 500 participants in the UK. .

Research 246
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FDA approves first gene therapy for Duchenne muscular dystrophy

Drug Discovery World

The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. Phase III EMBARK trial Consistent with the accelerated approval pathway, the company has committed to the completion of a confirmatory trial.

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FDA approves RSV vaccine to protect babies from birth

Drug Discovery World

The US Food and Drug Administration (FDA) has approved the first vaccine for pregnant individuals to prevent respiratory syncytial virus (RSV) in infants. This approval provides an option for healthcare providers and pregnant individuals to protect infants from this potentially life-threatening disease.”