NewsRSS3 days ago
This article invites people to take a quiz about clinical trials for HDFN (hemolytic disease of the fetus and newborn), which is a serious blood condition that affects unborn babies and newborns. After completing the quiz, you can see how your answers compare to other people who took it. The article doesn't provide new trial information, but rather encourages learning about existing trials through an interactive quiz.
WHY IT MATTERSTaking this quiz helps patients and caregivers understand what clinical trials are available for HDFN, which is important since treatment options and trial availability can directly affect care decisions for affected pregnancies and newborns.
NewsRSS3 days ago
This article is a quiz that tests what you know about riociguat, a medication used to treat pulmonary arterial hypertension (PAH). PAH is a rare condition where blood vessels in the lungs become narrowed, making it harder for the heart to pump blood through them. The quiz helps patients and caregivers learn more about how this treatment works.
WHY IT MATTERSUnderstanding how riociguat works and its role in PAH treatment can help patients make informed decisions about their care and have more meaningful conversations with their doctors about their treatment options.
NewsRSS3 days ago
This article invites people to take a quiz about life expectancy in AAV (ANCA-associated vasculitis), a rare disease that affects blood vessels. After completing the quiz, you can see how your answers compare to other people who took it. The quiz is a way to learn more about what to expect with this condition.
WHY IT MATTERSUnderstanding life expectancy and prognosis in AAV can help patients and caregivers make informed decisions about treatment options and long-term planning.
NewsRSS3 days ago
A tribute was published honoring Timothy Chan, a young man with Duchenne muscular dystrophy who recently passed away from a respiratory illness. The article celebrates how he inspired others through his approach to life, leadership, and compassion. Duchenne muscular dystrophy is a serious genetic condition that weakens muscles over time, and respiratory complications are a common concern for people with this disease.
WHY IT MATTERSThis story highlights the real-world impact of Duchenne muscular dystrophy and the importance of respiratory monitoring and care, which is critical for patients with this progressive condition.
AdvocacyRSS3 days ago
A mother shares her personal story about raising two sons with hemophilia, a bleeding disorder that prevents blood from clotting properly. She describes how the condition has affected her family's life and what it means to be a caregiver for young men living with this lifelong disease. The article uses her childhood memory of wanting to do gymnastics as a contrast to the physical limitations and challenges her sons face.
WHY IT MATTERSParents and caregivers of people with hemophilia can find community and validation in hearing directly from other families navigating the same daily challenges of managing a chronic bleeding disorder.
ResearchRSS3 days ago
Scientists discovered that blocking a protein pathway called JNK in the body may help treat spinal muscular atrophy (SMA), a disease that weakens muscles. In mice with severe SMA, blocking this pathway reduced how bad the disease got and helped the mice live longer. This new approach could work by itself or combined with medicines that are already approved for SMA.
WHY IT MATTERSIf this JNK pathway blocking approach moves to human trials, it could offer SMA patients a new treatment option, potentially as a combination therapy with existing approved drugs like nusinersen or onasemnogene abeparvovec.
NewsRSS3 days ago
A person living with ALS shares their experience learning to use ankle-foot orthoses (AFOs), which are braces that help with walking. At first, they didn't want to use them because they represented another big life change after their ALS diagnosis. But through trial and error, they found ways to adapt and make the braces work for them.
WHY IT MATTERSThis personal account offers practical insights into managing mobility challenges with ALS, which can help newly diagnosed patients understand what to expect and how others have successfully adapted to assistive devices.
Clinical trialRSS3 days ago
A new drug called masitinib helped ALS patients live longer than expected. In a clinical trial, over 40% of patients treated with masitinib survived for at least 5 years from when their disease started. This is much better than the typical outcome, where fewer than 25% of ALS patients usually live that long.
WHY IT MATTERSIf masitinib gains approval, ALS patients may have access to a treatment that could significantly extend their survival time compared to current standard care.
NewsRSS3 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.
AdvocacyRSS3 days ago
This article announces a support group meeting in Bethesda, Maryland for people with rare diseases. Support groups bring together patients and caregivers to share experiences, get information, and find emotional support from others facing similar health challenges. The meeting was scheduled for Saturday, July 17, 2027.
WHY IT MATTERSSupport groups provide patients with rare diseases a chance to connect with others who understand their condition and learn practical coping strategies from people with lived experience.
AdvocacyRSS3 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, get information, and find emotional support from others dealing with the same condition.
WHY IT MATTERSThis support group provides a local community resource for MG patients in Oregon and Washington to connect with others managing this rare autoimmune disease and access peer support.
AdvocacyRSS3 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.
AdvocacyRSS3 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 Northeastern Pennsylvania. Support groups bring together patients and caregivers to share experiences, get information, and find emotional support from others dealing with the same condition.
WHY IT MATTERSThis support group provides a local community resource for MG patients in Northeastern PA to connect with others, share coping strategies, and access peer support—which research shows improves quality of life and treatment adherence.
AdvocacyRSS3 days ago
This is an announcement for a support group meeting for people with myasthenia gravis (MG) in the Sacramento-Sierra region of California. The group meets on Saturday mornings and provides a space for patients and caregivers to connect with others who have the same condition. Support groups help people share experiences, get information, and feel less alone.
WHY IT MATTERSThis local support group offers Sacramento-area MG patients a chance to connect with others managing the same autoimmune condition and access peer support that can improve quality of life and disease management.
AdvocacyRSS3 days ago
A support group for people with myasthenia gravis (MG) — a rare condition where the immune system weakens muscles — is meeting in the Hampton Roads area of Virginia. Support groups bring together patients and caregivers to share experiences, learn from each other, and find emotional support from others facing the same condition.
WHY IT MATTERSThis local support group provides Hampton Roads patients with MG a chance to connect with others managing the same rare disease, access peer support, and potentially learn about treatment options and coping strategies from people with lived experience.
AdvocacyRSS3 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.
AdvocacyRSS3 days ago
This is an announcement for a Myasthenia Gravis (MG) support group meeting in the Sacramento-Sierra region of California. The group meets on Saturday mornings at 10:00 AM. Support groups bring together people with the same condition to share experiences, ask questions, and find community support.
WHY IT MATTERSThis support group provides MG patients in Northern California a local opportunity to connect with others managing the same autoimmune condition and access peer support and shared resources.
AdvocacyRSS3 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.
AdvocacyRSS3 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 Hampton Roads area of Virginia. Support groups bring together patients and caregivers to share experiences, get information, and find emotional support from others dealing with the same condition.
WHY IT MATTERSIf you or a loved one has myasthenia gravis in the Hampton Roads area, this support group offers a chance to connect with others managing the same rare condition and access local resources and peer support.
AdvocacyRSS3 days ago
A support group for people with myasthenia gravis (MG), a rare condition where the immune system weakens muscles, is meeting in Northeastern Pennsylvania. Support groups bring together patients and caregivers to share experiences, get information, and find emotional support from others dealing with the same condition.
WHY IT MATTERSThis support group provides a local community resource for MG patients in Northeastern PA to connect with others managing this rare autoimmune condition and access peer support.