A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.

Families began receiving calls from doctors last week to arrange treatments for new Givinostat drug after a campaign by the ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

Mom, us children decided to each write a birthday message to you for your 54th birthday, May 22. Sitting here, dozens of ...

Lovina turned 54 years old on May 22. Her children decided to write a tribute column for her. Each child will write a few ...

The "Duchenne Muscular Dystrophy - Global Clinical Trials Review, 2025" report provides a comprehensive overview of the global clinical trials landscape for Duchenne Muscular Dystrophy. It includes ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

Leah Messeropened up to OK! about rumors the show is ending, her relationship with her former Teen Mom 2costars and an update ...

The Idaho Falls Fire Department is once again upholding a cherished tradition, partnering with the Muscular Dystrophy Association (MDA) for their annual "Fill the Boot" fundraising campaign this ...

Jo has been holding these fundraising plant sales annually for several years, with each sale contributing to the charity's efforts.

Jonathan Strober, MD, explains the initial presentation of myasthenia gravis in a pediatric patient population and why initial diagnosis for these patients can be challenging.