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.

FDA Rejects Regeneron’s Blood Cancer Therapy for Two Forms of Lymphoma 3/25/2024

Novo’s $1.1B Cardior Buy Continues Expansion into Heart Disease 3/25/2024

AbbVie Acquires Landos in Potential $212M Deal, Bolsters Anti-Inflammatory Pipeline 3/25/2024

J&J Wins FDA Nod for Pulmonary Arterial Hypertension Combo Pill 3/25/2024

FDA Authorizes Invivyd’s Pemgarda to Prevent COVID-19 in Immunocompromised People 3/25/2024

AbbVie Secures Full FDA Approval for ADC Elahere in Ovarian Cancer 3/25/2024

Esperion Targets Seven-Fold Jump in Cholesterol Drug Population with FDA Label Expansion 3/25/2024

Wegovy Cardiovascular Approval Furthers March into New Indications 3/25/2024

Epigenetic Editing Explodes on the Heels of Gene Editing Success 3/25/2024

Merck, Others Look to Immunotherapy Combos as Next Frontier in Oncology 3/25/2024

Opinion: ALS Is Not a Singular Disease. Stop Treating It Like One 3/25/2024

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.

FDA Approves Third Duchenne Muscular Dystrophy Treatment in Nine Months 3/22/2024

Medicare to Cover Novo’s Weight-Loss Drug Wegovy for Patients with Heart Disease 3/22/2024

Eledon’s Antibody Aids First Gene-Edited, Pig-to-Human Kidney Transplant 3/22/2024

Roche’s IL-6 Blocker Clears Phase III Bar, Still Falls Short of Expectations 3/22/2024

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.