Clinical trialRSSToday
A grandmother shares how a small change in how her grandson's condition was measured helped him qualify for a Duchenne muscular dystrophy (DMD) treatment trial. Researchers adjusted the scoring system by 19 points, which gave him a second chance to participate in the study. This story highlights how trial eligibility rules can sometimes be adjusted to help more patients access new treatments.
WHY IT MATTERSIf your child with DMD has been told they don't qualify for a trial, this story shows that eligibility criteria can sometimes be reconsidered or adjusted—it's worth asking your doctor whether similar flexibility might apply to your situation.
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.
ResearchRSSToday
A new study found that babies born early who develop a lung condition called bronchopulmonary dysplasia (BPD) often need medications to treat high blood pressure in their lungs (pulmonary hypertension). However, as these children grow into toddlers around age 2, many of them stop needing these medications. This suggests that the lung condition may improve naturally as kids get older.
WHY IT MATTERSIf your child with BPD and pulmonary hypertension is on PH medications, this research suggests their doctor may be able to reduce or stop treatment as they grow, potentially reducing long-term medication burden and side effects.
Clinical trialRSSToday
A new treatment called Roctavian was given as a single injection to men with severe hemophilia A (a bleeding disorder). Seven years later, most patients had fewer bleeding episodes and needed less preventive medicine. This suggests the treatment's benefits can last for many years.
WHY IT MATTERSIf you have severe hemophilia A, Roctavian could mean switching from frequent infusions or injections to a single treatment that protects you from bleeds for years.
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.
PolicyRSSToday
President Trump signed an executive order on April 18 directing the federal government to speed up access to treatments for serious mental illness, including conditions that are hard to treat. This means the FDA and other health agencies will work faster to get new mental health treatments to patients who need them.
WHY IT MATTERSPatients with treatment-resistant mental illness conditions may gain faster access to new therapies through streamlined FDA review processes and accelerated approval pathways.
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.
🔴 BreakingDrug approvalRSSYesterday
The FDA has approved Otarmeni, a new gene therapy treatment for genetic hearing loss. This is the first-of-its-kind treatment that uses a special virus to deliver healthy genes into the ear to help restore hearing. The approval was fast-tracked through a special FDA program that prioritizes treatments for serious diseases.
WHY IT MATTERSThis is the first gene therapy approved specifically for inherited hearing loss, offering a potential one-time treatment option for patients with certain genetic forms of deafness who previously had no curative options.
ResearchRSSYesterday
A large study found that children with Duchenne muscular dystrophy (DMD) who started taking corticosteroids—a type of anti-inflammatory medicine—earlier in life had better muscle strength and movement than children who started the medicine about a year later. This shows that starting treatment as soon as possible after diagnosis helps preserve how well kids can walk, run, and use their muscles.
WHY IT MATTERSIf you have a young child with DMD, this research provides strong evidence to discuss with your doctor about starting corticosteroid treatment immediately after diagnosis rather than waiting, as earlier treatment may significantly slow muscle weakness.
NewsRSS2 days ago
A patient with AATD (a rare lung disease caused by low levels of a protective protein) shares her personal experiences with complementary and alternative medicine approaches. She discusses how nontraditional treatments have fit into her overall care plan alongside standard medical treatment. The article reflects one patient's perspective on combining different types of medicine to manage her condition.
WHY IT MATTERSPatients with AATD can learn from one person's real-world experience balancing conventional treatment with complementary approaches, which may help inform their own conversations with their healthcare team about what options exist.
Clinical trialRSS2 days ago
A company called BlueRock Therapeutics tested a new cell therapy called bemdaneprocel for Parkinson's disease. In a small early-stage trial, patients who received one dose of this therapy showed improvement in their movement symptoms, and these improvements lasted for up to three years. This is promising because it suggests a single treatment might help Parkinson's patients for a long time.
WHY IT MATTERSIf bemdaneprocel continues to show sustained benefits in larger trials, it could offer Parkinson's patients a one-time treatment option instead of daily medications, potentially improving quality of life and treatment adherence.
AdvocacyRSS2 days ago
A patient with MS shares their personal experience of why starting treatment quickly with the most powerful available medications is worth it to them. They believe the benefits of aggressive, early treatment outweigh the potential side effects. This is one patient's perspective on MS treatment strategy.
WHY IT MATTERSThis patient perspective highlights the real-world decision-making that people with MS face when choosing between starting strong treatments early versus waiting, which can help other newly diagnosed patients think through their own treatment conversations.
NewsRSS2 days ago
This article is a quiz about nerandomilast, a medicine being studied to treat progressive pulmonary fibrosis (PPF), which is a lung disease where lung tissue becomes scarred and stiff over time, making it harder to breathe. The quiz helps patients and caregivers learn more about this potential treatment option. It's a way to test your understanding of how this medicine might help people with this serious lung condition.
WHY IT MATTERSPatients with progressive pulmonary fibrosis have limited treatment options, so learning about emerging therapies like nerandomilast helps them stay informed about potential new ways to slow lung damage.
NewsRSS2 days ago
Children with Duchenne muscular dystrophy (DMD) need physical therapy to stay mobile as long as possible, but doctors and families are trying to figure out how to balance therapy with letting kids just be kids and play. The article explores how to make treatment feel less like a chore and more like a normal part of childhood.
WHY IT MATTERSFamilies managing DMD face a real tension between following medical recommendations for physical therapy and allowing their children to enjoy normal childhood activities—this article addresses that specific struggle.
NewsRSS2 days ago
A person who was diagnosed with limb-girdle muscular dystrophy 30 years ago finally took a genetic test to find out exactly which gene mutation caused their condition. Genetic testing can help doctors understand the specific type of muscular dystrophy a patient has, which may affect treatment options and family planning decisions. This story shows how genetic testing technology has improved over time and can provide answers even decades after an initial diagnosis.
WHY IT MATTERSGenetic confirmation of limb-girdle muscular dystrophy can unlock access to gene-specific clinical trials and emerging therapies that weren't available at the time of initial diagnosis.
AdvocacyRSS2 days ago
A parent shares their experience with their son's delayed diagnosis of thymidine kinase 2 deficiency (TK2d), a rare genetic disorder affecting how the body uses certain building blocks for DNA. The article highlights how early genetic testing could have identified the condition sooner and made a significant difference in the child's care and outcomes. Early testing is important because it allows doctors to start treatment and management strategies before serious complications develop.
WHY IT MATTERSParents of children with unexplained developmental delays or muscle weakness can use this story to advocate for early genetic testing with their doctors, potentially catching TK2d before irreversible damage occurs.
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 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.
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
When you have Parkinson's psychosis, having a care team that works together can make a big difference. A coordinated care team helps manage symptoms better, makes sure doctors and patients talk clearly with each other, and helps you and your family make better decisions about treatment. This approach brings together different specialists to support you in one organized way.
WHY IT MATTERSParkinson's psychosis patients often see multiple doctors separately; a coordinated care team ensures all providers share information and work toward the same treatment goals, reducing confusion and improving symptom management.