ResearchRSS3 days ago
A new study shows that regular exercise — including activities like walking, strength training, and balance work — is safe and helpful for people with hemophilia of all ages. The research found that exercise can reduce chronic pain, improve how well people can move and function, and make life better overall. The key is that exercise programs need to be carefully designed for each person's needs.
WHY IT MATTERSPeople with hemophilia often avoid exercise due to bleeding concerns, but this study provides evidence that structured exercise can safely reduce pain and improve daily functioning without increasing bleeding risk.
ResearchRSS3 days ago
A new study used wrist sensors to track physical activity in children with pulmonary hypertension (a condition where blood pressure in the lungs is too high). Researchers found that kids with this condition exercise less intensely and for shorter periods than healthy children. Children with more severe cases had even bigger drops in exercise intensity.
WHY IT MATTERSUnderstanding how pulmonary hypertension limits children's physical activity can help doctors and families set realistic exercise goals and monitor whether treatments are helping kids stay more active.
NewsRSS3 days ago
Orenitram is a pill form of a medicine called treprostinil that helps people with pulmonary arterial hypertension (a serious lung condition where blood vessels in the lungs become too narrow). You take it by mouth 2-3 times a day with food. This medicine helps patients exercise better and slows down how fast their disease gets worse.
WHY IT MATTERSThis oral option gives PAH patients an alternative to injected or inhaled treprostinil, making treatment more convenient for daily management.
Clinical trialUNITERAREApr 3
Researchers are testing a new technology called 'digital twins' to help prevent second strokes in patients with three rare brain conditions: moyamoya disease, cerebral amyloid angiopathy, and previous stroke history. A digital twin is a computer model of a patient's brain that doctors can use to predict what treatments might work best for that individual. This study will randomly assign patients to either receive care guided by this digital twin technology or standard care, and compare which approach prevents more strokes.
WHY IT MATTERSThis trial offers patients with moyamoya disease or cerebral amyloid angiopathy who have had a stroke access to personalized, AI-guided prevention strategies that could reduce their risk of future strokes.
Clinical trialCLINICALTRIALSMar 26
Researchers are testing a new way to help young people with rare bone diseases get better. The program combines sailing therapy (being out on the water) with online rehabilitation exercises that patients can do at home. This study builds on earlier successful results and will compare two different treatment approaches to see which works best.
WHY IT MATTERSThis trial offers young patients with rare skeletal disorders access to an innovative rehabilitation model combining in-person sailing therapy with remote maintenance care, potentially improving outcomes beyond traditional therapy alone.