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 ...

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 ...

Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD.

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 ...

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular ...

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

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 ...