PolicyRSS2 days ago
A large survey in the U.K. found that most people—including families of babies screened for SMA, people living with SMA, doctors, and the general public—strongly support newborn screening for spinal muscular atrophy. The main reason people liked the idea was that catching SMA early could help babies have better health outcomes. Researchers looked at what factors influenced people's opinions about this type of screening.
WHY IT MATTERSIf newborn SMA screening becomes standard in the U.K., babies diagnosed early could start treatment sooner, potentially preventing severe muscle weakness and improving their long-term quality of life.
AdvocacyRSS4 days ago
The PBC Foundation International Summit is a conference where people living with primary biliary cholangitis (PBC)—a rare liver disease—share their experiences and perspectives. Unlike typical medical conferences focused mainly on doctors and research, this summit puts patients' voices at the center of discussions. A woman with PBC explains what makes this event different and valuable for the community.
WHY IT MATTERSPatient-centered conferences like this one help people with PBC connect with others, learn directly from their peers' experiences, and ensure their needs shape future research and treatment discussions.
AdvocacyRSS4 days ago
This article uses the excitement of NASA's Artemis II space mission as inspiration to encourage big dreams about finding a cure for pulmonary hypertension, a serious lung disease that affects blood vessels. The author draws a parallel between humanity's ability to reach for the stars and the hope that patients with pulmonary hypertension should have for breakthrough treatments. The piece is motivational rather than reporting on specific medical news or research developments.
WHY IT MATTERSThis article may resonate emotionally with pulmonary hypertension patients by framing their disease journey within a larger narrative of human achievement and possibility, though it does not announce any new treatments, trials, or clinical developments.