ResearchPUBMEDToday
Researchers in Italy studied how rare diseases affect older adults. They found that thousands of people are diagnosed with rare diseases after age 65, and thousands more who were diagnosed as children or young adults are now living into old age with these conditions. This study shows that rare diseases in elderly people are becoming more common and important to understand.
WHY IT MATTERSIf you or a family member has a rare disease and are aging, this research highlights that healthcare systems need better plans to care for older patients with rare conditions—which could lead to improved treatment strategies and support services tailored to your needs.
NewsRSSToday
A person with Parkinson's disease shares their experience living well with the condition after receiving deep brain stimulation surgery. They started a weekly walking group three years ago to stay active and connected with others. The article focuses on practical tips and lifestyle strategies for managing Parkinson's disease day-to-day.
WHY IT MATTERSThis firsthand account demonstrates how deep brain stimulation combined with regular physical activity and social engagement can help people with Parkinson's disease maintain quality of life and independence.
Clinical trialRSSToday
A new experimental drug called tavapadon from AbbVie helped people with early Parkinson's disease delay or avoid starting levodopa, which is the standard treatment for this condition. In a completed Phase 3 trial called TEMPO-4, most people taking tavapadon long-term did not need to start levodopa, and those already taking it didn't need to increase their dose. This suggests tavapadon could be a helpful new option for managing early Parkinson's symptoms.
WHY IT MATTERSIf approved, tavapadon could allow people with early Parkinson's to delay levodopa therapy, potentially postponing side effects that come with long-term use of this standard medication.
NewsRSSYesterday
A person with SMA (spinal muscular atrophy) experienced a serious fall onto concrete while their nurse was helping them get dressed, resulting in intense knee pain. The article discusses how this unexpected injury was a frightening experience for someone already managing a complex rare disease. The post appears to be a personal account about dealing with this new complication.
WHY IT MATTERSPeople with SMA have increased fall risk and fragility due to muscle weakness, making even routine activities like being dressed potentially dangerous — this story highlights a real safety concern for SMA patients and caregivers.
AdvocacyRSS2 days ago
Children with Prader-Willi Syndrome (PWS) have real character strengths and positive qualities, but the challenges of the condition can make these strengths difficult for families to notice and support. Recognizing these strengths is important for family wellbeing. However, even when caregivers understand their child's strengths, actually supporting them can be exhausting for parents and guardians who are already managing the demands of PWS.
WHY IT MATTERSFor families managing PWS, understanding that their child has genuine strengths—beyond the condition's challenges—can improve how they view their child and their own emotional wellbeing, even though caregiving remains demanding.
NewsRSS2 days ago
A patient with AATD (Alpha-1 Antitrypsin Deficiency) found personal inspiration by watching the movie Top Gun: Maverick, discovering parallels between the fighter pilot's perseverance and their own journey managing a chronic lung disease. The article explores how popular culture can provide emotional support and motivation for people living with rare diseases. This is a personal reflection piece rather than medical news.
WHY IT MATTERSFor AATD patients, finding relatable stories and sources of inspiration in everyday media can help combat the isolation and emotional burden of managing a progressive genetic lung disease.
NewsRSS2 days ago
A patient with ATTR-CM (a rare heart disease caused by abnormal protein buildup) is sharing their experience saying goodbye to a doctor who understood their condition deeply. When you have a rare disease, finding a doctor who takes time to learn your personal story and medical history is especially valuable. This article highlights how important that doctor-patient relationship is for people managing uncommon health conditions.
WHY IT MATTERSFor ATTR-CM patients, continuity of care with a knowledgeable physician directly impacts treatment decisions and quality of life, making transitions to new doctors particularly challenging.
AdvocacyRSS2 days ago
People with pulmonary hypertension (PH) often feel pressure to push through exhaustion and symptoms like chest pain, but this approach can actually make their condition worse. The article explains that rest and pacing activities is important for managing PH, rather than trying to power through difficult days. Ignoring your body's signals can lead to more frustration and health problems.
WHY IT MATTERSThis article addresses a critical but often overlooked aspect of PH management: understanding that rest is medical treatment, not laziness, which can help patients make better decisions about activity and reduce unnecessary symptom flare-ups.
NewsRSS2 days ago
This article invites people with HAE (hereditary angioedema) to take a quiz about the different doctors and specialists who help care for HAE patients. You can compare your answers to what other people answered. It's a way to learn more about building a good care team for managing this condition.
WHY IT MATTERSUnderstanding which specialists to see—such as allergists, immunologists, and emergency doctors—helps HAE patients get better coordinated care and faster treatment when attacks happen.
AdvocacyRSS2 days ago
A patient with CIDP, a rare nerve disease that causes weakness and tingling, describes how much time the condition takes up each week—not just doctor visits, but also managing symptoms, doing physical therapy, and dealing with fatigue. The article shows that the real burden of living with CIDP goes beyond what doctors usually measure, including all the hidden time spent managing the disease day-to-day.
WHY IT MATTERSUnderstanding the time burden of CIDP helps patients feel less alone in their struggles and gives them language to explain to family, employers, and doctors why the disease affects their quality of life so much.
AdvocacyRSS2 days ago
This article offers practical advice for people with hereditary angioedema (HAE) who feel limited by their condition. The author encourages patients to focus on what they can do rather than what they can't, and suggests ways to live a full life while managing HAE symptoms.
WHY IT MATTERSHAE patients often experience social isolation and emotional burden from unpredictable swelling attacks; this article addresses the psychological impact and provides coping strategies to improve quality of life.
NewsRSS2 days ago
ATTR-CM is a rare heart disease caused by abnormal protein buildup, but many patients also experience serious stomach and digestive problems that doctors don't always talk about. These gastrointestinal issues—like difficulty swallowing, nausea, and poor nutrient absorption—can be just as challenging as the heart symptoms and significantly impact quality of life. The article highlights that patients need better awareness and support for managing these hidden digestive complications alongside their heart treatment.
WHY IT MATTERSATTR-CM patients often suffer from overlooked gastrointestinal complications that can worsen malnutrition and quality of life, yet these symptoms receive less medical attention than cardiac manifestations—understanding this connection helps patients advocate for comprehensive care.
NewsRSS2 days ago
When you have a rare disease like Alpha-1 Antitrypsin Deficiency (AATD), taking care of your mental health is just as important as managing your physical symptoms. The article explains that doing things you enjoy—like visiting aquariums or crocheting—can help reduce stress and improve your overall well-being. Having fun and finding joy in daily activities is a key part of living well with a rare disease.
WHY IT MATTERSAATD patients often face isolation and emotional burden from their condition, so this article directly addresses how mental health support through enjoyable activities can improve quality of life alongside medical treatment.
NewsRSS2 days ago
A person living with SMA (spinal muscular atrophy) discusses how having up-to-date technology is important for their daily life and work, not just a luxury. The article uses the metaphor of an aging laptop needing frequent repairs to illustrate how people with SMA may need newer devices to help them stay productive and independent. The piece highlights that technology access is a practical health and quality-of-life issue for people managing this condition.
WHY IT MATTERSPeople with SMA often rely on assistive technology and adaptive devices to work and participate in daily activities, making access to current, reliable technology a functional health necessity rather than a luxury.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system weakens muscles, is meeting in St. Petersburg, Florida. Support groups bring together patients and caregivers to share experiences, ask questions, and learn from each other about managing their condition.
WHY IT MATTERSIf you or a loved one has myasthenia gravis in the St. Petersburg area, this support group offers a chance to connect with others facing the same challenges and access local resources.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system weakens muscles, is meeting in the Pacific Northwest region covering Oregon and Washington. Support groups bring together patients and caregivers to share experiences, learn from each other, and find emotional support. This group provides a local community resource for people managing this chronic condition.
WHY IT MATTERSLocal support groups help MG patients connect with others facing the same challenges, reduce isolation, and access peer knowledge about managing symptoms and navigating treatment options in their region.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system attacks muscles and causes weakness, is meeting in St. Petersburg, Florida. Support groups bring together patients and caregivers to share experiences, ask questions, and learn from each other about managing their condition.
WHY IT MATTERSIf you or a loved one has myasthenia gravis in the St. Petersburg area, this support group offers a chance to connect with others facing the same challenges and get practical advice from people living with the condition.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system weakens muscles, is meeting in St. Petersburg, Florida. Support groups bring together patients and caregivers to share experiences, ask questions, and learn from each other about managing their condition.
WHY IT MATTERSIf you or a loved one has myasthenia gravis in the St. Petersburg area, this support group offers a chance to connect with others facing the same challenges and access local resources.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system weakens muscles, is meeting in St. Petersburg, Florida. Support groups bring together patients and caregivers to share experiences, ask questions, and learn from each other about managing their condition.
WHY IT MATTERSThis local support group provides St. Petersburg-area MG patients with a chance to connect with others facing the same disease, access peer support, and potentially learn about new treatments or clinical trials from group members.
AdvocacyRSS2 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system attacks muscles and causes weakness, is meeting in the Pacific Northwest region covering Oregon and Washington. Support groups bring together patients and caregivers to share experiences, learn from each other, and find emotional support. This group provides a local community resource for people managing this chronic condition.
WHY IT MATTERSThis support group offers MG patients in Oregon and Washington a chance to connect with others facing the same condition, which can reduce isolation and provide practical tips for managing symptoms and treatment.