Data from the Phase Ia/b study demonstrated that SAT-3247 was well-tolerated and safe with a desirable PK profile.

NS Pharma, Inc. (NS Pharma), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that the National Center of Neurology and ...

Facioscapulohumeral Muscular Dystrophy (FSHD) is a genetic disorder marked by muscle weakness, primarily in the face, shoulders, and arms. The DUX4 gene mutation leads to toxic protein production, ...

Approximately 16 boys with Duchenne Muscular Dystrophy (DMD), including several teenagers, are set to benefit from the drug, givinostat. Patients will have to meet certain criteria, including being ...

Sarepta says it has what could be the first gene therapy for limb girdle muscular dystrophy. But approval is going to be ...

Find out if it’s time to ask about your child’s symptoms — and if they could be a sign of Duchenne. Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that ...

Medically reviewed by Brigid Dwyer, MD Key Takeaways Life expectancy for muscular dystrophy depends on the type and severity ...

Muscular Dystrophies are a group of genetic conditions characterized by muscle weaknesses. They are genetic conditions and have different sub-categories depending on which area of muscle mass is ...

Long-term treatment with givinostat in boys with Duchenne muscular dystrophy continues to show a favourable safety and tolerability profile ...

Families caring for individuals with Duchenne muscular dystrophy (DMD) face significant financial burdens from necessary home and vehicle modifications to enhance quality of life. Artificial ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...