The U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
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Some anti-cancer treatments not only target tumour cells but also healthy cells. If their effects on the latter are too strong, their use can become limiting. A team from the University of Geneva (UNIGE), in collaboration with Basel-based FoRx Therapeutics, has identified the mechanism of action of PARP inhibitors, used in particular for breast and ovarian cancer in patients carrying the BRCA gene mutation.
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The Time Has Come to Make Weight-Loss Drugs Available to Those Who Need Them 3/22/2024
Bayer’s Prostate Cancer Drug Nubeqa on Track to Generate Blockbuster Sales in 2024 3/22/2024
An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH Clinical Center and then followed for about six years to assess safety.