To better understand patient experiences, the researchers analyzed qualitative and quantitative data from their social media ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...

Treatment guidelines in polycythemia vera currently recommend maintaining hematocrit below 45%, with a higher threshold for ...

Karen Winkfield, MD, PhD, addresses the importance of meeting patients where they are to expand clinical trial participation ...

Measles can be prevented with the measles-mumps-rubella (MMR) vaccine, which is recommended for children at 12 to 15 months ...

Celiac disease can lead to both disease-related and diet-induced vitamin deficiencies, making it crucial for patients to ...

Tania Gendron, PhD, speaks to the present challenges for translating biomarker discoveries to clinical practice and offers ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...

Researchers also highlighted the significance of TTNT-D as a real-world measure of treatment efficacy, noting it provides ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Annie Antar, MD, PhD, discusses how patients with HIV can have different and longer-lasting symptoms of COVID-19.

Leigh Maria Ramos Platt, MD, Children's Hospital Los Angelas, is also a medical director for the Muscular Dystrophy Association Neuromuscular Clinic. Her research focus includes neuromuscular ...