Clinical trialCLINICALTRIALSToday
Researchers are testing a new drug called BH-30643 for advanced lung cancer patients whose tumors have specific genetic mutations (EGFR or HER2). The study will first figure out the right dose and watch for side effects, then test how well the drug works against the cancer. About 266 patients will participate across multiple hospitals.
WHY IT MATTERSThis trial is now actively recruiting patients with EGFR and/or HER2-mutated advanced NSCLC — if you have this genetic profile and have exhausted standard treatments, you may be eligible to access a potentially new treatment option.
GrantRSSToday
The Muscular Dystrophy Association is holding its 26th annual fundraising gala in New York City on June 4 to raise money for ALS research. The event brings together financial leaders and medical researchers to support the search for treatments and cures for ALS, a disease that affects nerve cells controlling muscles.
WHY IT MATTERSFunding from this gala directly supports ALS research that could lead to new treatments, making it important for patients to know where research dollars are being invested and what progress is being made.
AdvocacyRSSToday
A caregiver shares her experience of learning to navigate life after her husband was diagnosed with ALS in 2018. She describes how she went from knowing almost nothing about the disease to becoming an active member of the ALS community. The article focuses on the emotional journey and finding support during a difficult time.
WHY IT MATTERSFor ALS caregivers and newly diagnosed patients, this personal account provides practical insights into building a support network and coping strategies during the early, overwhelming months after diagnosis.
NewsRSSToday
Some people with Parkinson's disease experience hallucinations or false beliefs, which is called psychosis. However, sudden changes in thinking or behavior might actually be caused by a urinary tract infection (UTI) rather than Parkinson's itself. Learning to tell the difference between these two conditions helps doctors give the right treatment.
WHY IT MATTERSParkinson's patients experiencing sudden confusion or hallucinations need to know that a UTI could be the culprit—and treating the infection might resolve symptoms without changing Parkinson's medications.
ResearchRSSYesterday
A study of 121 people with ALS found that those who kept their weight more stable after starting tube feeding lived longer. This was true even if they weren't eating a lot of calories when they first started the tube feeding. The finding suggests that preventing weight loss after tube feeding may help people with ALS live longer.
WHY IT MATTERSIf you or a loved one with ALS is considering or has started tube feeding, this research suggests that working with your care team to maintain steady weight—not just calorie intake—could potentially extend survival.
NewsRSSYesterday
This is a personal story about a man with ALS (a disease that affects nerve cells and causes muscle weakness) and how his dog Comet has become an important part of his life. As Todd's mobility has decreased over time, he's found creative ways to spend time with Comet, like riding on a scooter with the dog. The story shows how pets can bring joy and connection to people living with serious illnesses.
WHY IT MATTERSFor people with ALS and their caregivers, this story demonstrates how adaptive strategies and pet companionship can maintain quality of life and emotional well-being as the disease progresses.
AdvocacyRSS2 days ago
Bruce Campbell, who has Pompe disease, is competing in the Absa Cape Epic, one of the world's toughest mountain biking races. Despite living with a rare muscle disease that affects his strength and endurance, he decided to take on this extreme athletic challenge. His story shows how people with rare diseases can push their limits and achieve goals they set for themselves.
WHY IT MATTERSThis story demonstrates that people living with Pompe disease can maintain active lifestyles and pursue challenging physical goals, which may inspire patients to discuss realistic exercise options with their care team.
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.
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.
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
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.
NewsRSS2 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 trialRSS2 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.
🔴 BreakingDrug approvalRSS2 days ago
The FDA has approved an updated version of a wristwatch-like device called Cala kIQ Plus that helps reduce hand shaking in people with Parkinson's disease. This new version improves on the earlier Cala kIQ device. The device works by sending gentle electrical signals to help control tremor during movement.
WHY IT MATTERSPeople with Parkinson's disease who experience hand tremor during activities now have access to an improved wearable option that doesn't require surgery or daily medications.
NewsRSS2 days ago
A parent shares a personal story about receiving a song recording from their son with hemophilia, reflecting on how their child's talents and abilities extend far beyond his medical condition. The article emphasizes that people living with rare diseases like hemophilia are whole individuals with gifts and passions that deserve recognition. This piece appears in Hemophilia News Today and focuses on the emotional and human side of living with a chronic blood disorder.
WHY IT MATTERSFor parents and caregivers of children with hemophilia, this story validates the importance of seeing their child as a complete person with abilities and dreams, not just defined by their bleeding disorder diagnosis.
Clinical trialRSS2 days ago
A new medication called Vyalev helped people with Parkinson's disease stop experiencing unwanted, uncontrolled movements (called dyskinesia) when they switched to it from other Parkinson's drugs. In two clinical trials, most people saw these troublesome movements go away within a few months of starting Vyalev. This is good news because dyskinesia is a common side effect that happens after taking Parkinson's medications for a long time.
WHY IT MATTERSIf you have Parkinson's disease and experience dyskinesia from your current levodopa treatment, switching to Vyalev could eliminate these involuntary movements within months rather than managing them as a permanent side effect.
ResearchRSS2 days ago
A study from Japan found that people with pulmonary hypertension (a condition where blood vessels in the lungs become stiff and narrow) who have higher pulmonary vascular resistance—a measure of how hard the heart has to work to pump blood through the lungs—tend to have worse health outcomes. The study also looked at whether a type of diabetes medication called SGLT2 inhibitors might help these patients.
WHY IT MATTERSIf you have pulmonary hypertension related to heart disease, knowing that high pulmonary vascular resistance predicts worse outcomes could help your doctor decide whether to start more aggressive treatment earlier or monitor you more closely.
PolicyRSS2 days ago
Doctors are now recommending that all ALS patients get genetic testing to understand if their disease is inherited. A genetic counselor is a specialist who helps explain what these test results mean and how they might affect family members. Understanding your genetic information can help you and your doctor make better decisions about your care and family planning.
WHY IT MATTERSIf you have ALS, genetic testing and counseling can reveal whether your condition is hereditary, which affects whether your relatives should be screened and what treatment options might work best for you.
NewsRSS2 days ago
An ALS caregiver shares how reading a novel about a woman with cancer helped her process difficult emotions and questions she faces while caring for her husband who has had ALS for 16 years. The article explores how literature can be a meaningful tool for caregivers dealing with serious illness in their families. It highlights the emotional and psychological challenges that come with long-term caregiving.
WHY IT MATTERSALS caregivers often experience isolation and emotional burden — this article validates that seeking emotional support through creative outlets like reading is an important part of managing caregiver wellbeing.
AdvocacyRSS2 days ago
Three organizations that work with facioscapulohumeral muscular dystrophy (FSHD) patients are joining together to make clinical trials better. FSHD is a rare muscle disease that causes weakness in the face, shoulders, and upper arms. This partnership wants to improve how these trials are designed so they can test new treatments more effectively.
WHY IT MATTERSBetter-designed clinical trials mean faster progress toward treatments for FSHD, and patient input through the FSHD Society ensures trials are structured in ways that actually work for people living with the disease.