Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

8 articles from the last 90 days matching "movement problems"

Clinical trialRSSMay 15

Phase 3 trial of solengepras for Parkinson’s disease now fully enrolled

A large clinical trial testing a new Parkinson's disease medication called solengepras has finished enrolling 341 patients. This medication works differently than standard Parkinson's drugs and is being tested alongside regular treatments to see if it helps patients who experience movement problems that come and go throughout the day.

WHY IT MATTERSIf solengepras proves effective in this Phase 3 trial, it could offer Parkinson's patients a new treatment option that works through a different mechanism than current dopamine-based therapies, potentially helping those with motor fluctuations who don't respond adequately to standard medications alone.
👁 Watch this spaceParkinson's disease
ResearchRSSMay 14

Vyalev eases motor, nonmotor symptoms in advanced Parkinson’s

Vyalev is a new under-the-skin pump that delivers Parkinson's disease medication continuously. In a real-world study, it reduced the amount of time patients experienced symptoms and improved both movement problems and some other symptoms like mood or sleep issues. Patients also reported better quality of life.

WHY IT MATTERSIf you have advanced Parkinson's disease and struggle with medication wearing off between doses, Vyalev offers a continuous delivery option that may reduce symptom fluctuations and improve daily functioning.
💬 Ask your doctorParkinson's disease
GrantNIH REPORTERMay 11

New NIH Grant: The Impact of Environmental factors on Lewy Body Dementia: Combining Claims Data and Mechanistic Stu — $1.8M at Unknown Institution

Scientists are studying how things in our environment—like air pollution or chemicals—might increase the risk of Lewy body dementia, a brain disease that affects over one million Americans. Lewy body dementia happens when harmful proteins called alpha-synuclein build up in the brain and damage it, causing memory loss and movement problems. This $1.8 million research project will look at medical records and do lab studies to understand these environmental connections better.

WHY IT MATTERSIf researchers identify specific environmental triggers for Lewy body dementia, patients and families may be able to reduce their risk through lifestyle changes or protective measures.
Good to knowLewy body dementia
ResearchRSSMay 6

Genetic factors may hinder treatment in Parkinson’s caused by gene: Case

A woman with early-onset Parkinson's disease caused by a VPS13C gene mutation initially improved with medication and deep brain stimulation surgery, but then got much worse over time. Her motor symptoms (movement problems) and nonmotor symptoms (like mood or sleep issues) worsened rapidly, eventually making it impossible for her to live on her own. This case suggests that genetic causes of Parkinson's disease may affect how well standard treatments work.

WHY IT MATTERSIf you have VPS13C-related Parkinson's disease, this case shows that standard treatments like deep brain stimulation may not work as well long-term, so you should discuss genetic testing and personalized treatment plans with your neurologist.
💬 Ask your doctorParkinson's diseaseVPS13C-associated Parkinson's disease
Clinical trialUNITERAREMay 1

New Recruiting Trial: Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington's Disease

Researchers are testing a new treatment called low-intensity focused ultrasound that aims to help people with Huntington's disease by targeting a specific part of the brain called the pallidum. This non-invasive technique uses sound waves instead of surgery to potentially reduce movement problems and other symptoms. The trial is just starting in April 2026 and is looking for patients to participate.

WHY IT MATTERSThis is the first clinical trial testing focused ultrasound specifically for Huntington's disease pallidal treatment, offering patients a potentially non-invasive alternative to traditional deep brain stimulation surgery.
👁 Watch this spaceHuntington's disease
ResearchRSSApr 29

Gut protein misfolding may spot Parkinson’s years before symptoms

Researchers found that simple gut biopsies might detect signs of Parkinson's disease years before a person develops movement problems or other symptoms. They discovered that more than half of people with digestive issues but no diagnosed neurological disease showed protein misfolding in their gut tissue. This could allow doctors to identify people at high risk for Parkinson's much earlier than currently possible.

WHY IT MATTERSIf validated, routine gut biopsies could identify Parkinson's disease risk years before symptoms appear, potentially allowing earlier intervention when treatments might be most effective.
Good to knowParkinson's disease
Clinical trialCLINICALTRIALSApr 17

Trial Now Recruiting: A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease (NCT06826612)

Researchers are testing a new gene therapy called SPK-10001 to treat Huntington's disease, a rare brain disorder that causes movement problems and cognitive decline. This early-stage study will check if the treatment is safe and whether it helps patients. About 53 people with Huntington's disease will participate in this trial, which is being run by Roche, a major pharmaceutical company.

WHY IT MATTERSThis is one of the first human trials of SPK-10001, a gene therapy specifically designed to target the genetic cause of Huntington's disease, offering hope for a disease that currently has no cure.
You can act on thisHuntington's disease
Clinical trialUNITERAREApr 15

New Recruiting Trial: 18F-mFBG Cardiac Uptake With Lewy Body Dementia

Researchers are testing a new imaging scan called 18F-mFBG that can take pictures of the heart in people with Lewy body dementia, a brain disease that causes movement problems and thinking difficulties. This Phase 2 trial is now accepting patients and aims to see if this special scan can help doctors better understand and diagnose the disease. The scan uses a safe radioactive tracer that shows how well the heart's nerve endings are working.

WHY IT MATTERSThis trial is now actively recruiting patients with Lewy body dementia — if you have this diagnosis, you may be eligible to participate in a study that could help develop better diagnostic tools for your condition.
You can act on thisLewy body dementia

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases