AdvocacyRSSToday
A personal story about a man named Jared who has severe hemophilia B (a bleeding disorder) and seizures since childhood. The article highlights how teachers and classmates treated him as 'special' or different on his first day of fifth grade, showing how invisible illnesses can affect children socially and emotionally, not just physically.
WHY IT MATTERSChildren with hemophilia B and other invisible illnesses often face social stigma and misunderstanding in school settings, which can impact their mental health and quality of life as much as the medical condition itself.
PolicyRSS3 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.
AdvocacyRSS5 days ago
A patient with von Willebrand disease shares how much they depend on Humate-P, a medication made from donated plasma. The article highlights how plasma donors' generosity directly impacts the lives of people who need plasma-derived treatments to stay healthy and safe.
WHY IT MATTERSPatients relying on plasma-derived therapies like Humate-P depend on a consistent supply of donated plasma — understanding this connection may inspire more people to become plasma donors and help secure treatment availability.
AdvocacyRSS5 days ago
A patient-led nonprofit called The Speak Foundation has created a network of specialized clinics called LGMD Centers of Excellence to help people with limb-girdle muscular dystrophy (LGMD), a rare muscle disease. This new network aims to solve a major problem: patients with LGMD have had trouble getting consistent care and doctors have struggled to develop new treatments because the patient population is very small and spread out.
WHY IT MATTERSThis coordinated clinic network could help LGMD patients access specialized care in one place and speed up the development of new treatments by making it easier for researchers to find and study patients.
AdvocacyRSS5 days ago
Delphine Andrews, a life coach and disability advocate from North Carolina who lives with SMA (spinal muscular atrophy), shared her personal story in a podcast interview. She discussed how she learned to accept herself, help others with disabilities, and build a career as a life coach. The episode also covered important topics like managing caregivers and understanding how different parts of someone's identity intersect.
WHY IT MATTERSHearing from someone living with SMA about practical strategies for self-acceptance, caregiver management, and career transition can help newly diagnosed patients and families navigate similar challenges in their own lives.