PolicyRSSToday
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.
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.
ResearchRSS2 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.
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.
ResearchRSS3 days ago
Scientists studied how spinal muscular atrophy (SMA) damages the connections between nerves and muscles. They found that in people with SMA, these nerve-muscle connections don't develop properly right after birth, making them weak and unable to work well before nerve cells start dying. This early problem may be an important part of how SMA causes muscle weakness.
WHY IT MATTERSUnderstanding that nerve-muscle connection problems happen early in SMA—before nerve cell loss—could lead to new treatments that target this early stage rather than only treating later damage.
NewsRSS3 days ago
An adult with SMA (spinal muscular atrophy) who lives independently shares insights about how they built a caregiving team of over 20 volunteer friends instead of relying on paid professional caregivers. The article discusses strategies for finding and organizing friends to help with daily care needs, challenging the assumption that people with serious disabilities must use traditional caregiving services.
WHY IT MATTERSFor adults with SMA seeking independence, learning practical strategies to recruit and coordinate volunteer caregivers from your social network can make living on your own financially and socially sustainable.
NewsRSS3 days ago
The author shares their personal experience undergoing an overnight sleep study, comparing it to a previous study from 2002. They describe feeling nervous about the procedure but acknowledge that despite being uncomfortable, the study was necessary for their health. The article appears to be a patient perspective piece about the importance of sleep studies for diagnosis.
WHY IT MATTERSSleep studies are often critical diagnostic tools for patients with rare diseases like SMA (spinal muscular atrophy) that can affect breathing and sleep quality, making this firsthand account relevant to others considering or preparing for the same procedure.
ResearchRSS3 days ago
Scientists found that haloperidol, a medication normally used to treat psychiatric conditions, may help treat spinal muscular atrophy (SMA) by increasing levels of a protein called SMN that is missing or low in SMA patients. In laboratory tests with mouse cells and human patient cells, haloperidol helped nerve cells survive longer, reduced harmful inflammation, and improved movement. This suggests haloperidol could potentially be used alongside or instead of current SMA treatments.
WHY IT MATTERSIf haloperidol proves effective in human trials, SMA patients could potentially benefit from a medication that already exists and is FDA-approved, potentially offering a faster path to treatment than developing entirely new drugs.
NewsRSS3 days ago
A patient shares their experience moving from pediatric (children's) doctors to adult healthcare providers. The article describes the mixed feelings of relief and worry that come with this major life transition, using a waiting room scene to explore how gratitude and fear can happen at the same time.
WHY IT MATTERSTransitioning from pediatric to adult care is a critical moment for patients with rare diseases like SMA, where continuity of specialized treatment and emotional support can directly affect health outcomes and quality of life.
ResearchRSS3 days ago
A study of 34 patients in Hong Kong found that Evrysdi (a medicine taken by mouth) helped children and adults with spinal muscular atrophy (SMA) move better and feel better in their daily lives. Patients who had never taken SMA medicine before saw the biggest improvements. The good results lasted for up to three years.
WHY IT MATTERSThis real-world evidence from Asian patients shows Evrysdi works well outside of clinical trials, which may help doctors decide if it's the right treatment for SMA patients in your region.
NewsRSS3 days ago
This article discusses how people living with SMA (spinal muscular atrophy) can maintain healthy friendships with their caregivers, even when conflicts arise. The author emphasizes the importance of their caregiver community and explores ways to resolve disagreements while keeping these important relationships strong.
WHY IT MATTERSPeople with SMA depend on caregivers for daily support, making it crucial to know how to address conflicts constructively without damaging relationships that are essential to their health and independence.
AdvocacyRSS3 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.
ResearchRSS3 days ago
Researchers found that a simple electrical test using surface electrodes on the skin can measure nerve signals in people with spinal muscular atrophy (SMA). These signals were much weaker in SMA patients compared to healthy people, which suggests this test could be a useful way to track how the disease changes over time.
WHY IT MATTERSIf validated, this non-invasive surface electrode test could provide a faster, easier, and cheaper way to monitor SMA progression in clinical trials and patient care compared to current muscle strength assessments.
NewsUNITERARE4 days ago
The PAN Foundation's financial assistance program for people with Spinal Muscular Atrophy (SMA) is currently closed and not accepting new applications. The PAN Foundation helps patients pay for medications and treatment costs when they can't afford them. You can check the program website to see when it reopens or find other financial help options.
WHY IT MATTERSIf you have SMA and struggle to afford your treatment, knowing this fund is temporarily closed helps you explore alternative financial assistance programs before your medication runs out.
Clinical trialCLINICALTRIALSApr 9
Researchers at Columbia University are looking for 106 children and young adults with Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy Type 3 (SMA Type 3) to join a study. The study will use special sensors and technology to track how people walk and move in their daily lives over a long period of time. The goal is to find new ways to measure how these diseases are progressing so doctors can better understand and treat them.
WHY IT MATTERSThis trial is now actively recruiting participants with DMD and SMA Type 3, offering a chance to contribute to developing better measurement tools that could help doctors track disease progression and evaluate future treatments more accurately.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for spinal muscular atrophy (SMA) is currently closed and is not accepting new applications. This program previously provided up to $12,000 per year to help eligible patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have spinal muscular atrophy and need help paying for medications like Zolgensma or Spinraza, you'll need to explore other financial assistance programs since this major funding source is temporarily unavailable.