The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...

Vamorolone (Agamree) is available on the NHS as a possible treatment for Duchenne muscular dystrophy (DMD) in people 4 years and over. Is this treatment right for me? Your healthcare professionals ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

Some muscular dystrophies are mild. Others are more serious and can cause life-threatening muscle weakness. Most children with Duchenne muscular dystrophy ... weakness in the shoulders, upper ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 per year. Objectives: To quantify the magnitude of altruism value as applied to a ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle disease ... but also showed mild pseudohypertrophy of the calf muscles and abnormal increases in CK values that were not ...

The appearance of a new orca calf in Washington’s Puget Sound last month was cause for celebration. The young female, whom researchers named J61, was a new addition to the Southern Resident ...

This was not the mother’s first time losing a child. In 2018, the endangered orca known as Tahlequah carried the body of her dead calf for at least 17 days, traversing more than 1,000 miles of ...

On Jan. 1, 2025, an orca was spotted carrying her recently deceased calf in an apparent act of grief. The whale, also known as Tahlequah, had previously made headlines in 2018 for carrying her ...

Scientists believe Tahlequah is expressing grief by continuing to push the calf's body, a behavior that expends valuable energy. While other orcas have been observed carrying their dead ...