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FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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FDA panel votes against MDMA therapy for PTSD, citing muddy data and safety concerns

BioPharma Drive: Drug Pricing

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

FDA 130
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Meaningful Moment for HIV Treatment as FDA Approves ViiV’s Monthly Shot

The Pharma Data

Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare. Cabenuva reduces the treatment dosing days from 365 days to 12 days per year. Janssen is a Johnson & Johnson company.

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FDA Approves First COVID-19 Treatment for Young Children

The Pharma Data

This action makes Veklury the first approved COVID-19 treatment for children less than 12 years of age. As a result of today’s approval action, the agency also revoked the emergency use authorization for Veklury that previously covered this pediatric population. director of the FDA’s Center for Drug Evaluation and Research.

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Shifting Paradigms in PAH Clinical Trials: 7 Key Takeaways for Success

PPD

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma

The Pharma Data

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that the U.S.

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FDA Approves Updated Indication for Merck’s KEYTRUDA® (pembrolizumab) for Treatment.

The Pharma Data

In addition, as part of the label update, this indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for any platinum-containing chemotherapy.