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.
Clinical trialRSS2 days ago
A new clinical trial has started testing a drug called IKT-001 in people with pulmonary arterial hypertension (PAH), a rare condition where blood vessels in the lungs become narrowed and make it hard for the heart to pump blood. This is a large Phase 3 trial, which is a late-stage test that happens before a drug might be approved. Nearly 500 adults will participate to see if this drug works better than current treatments.
WHY IT MATTERSIf you have PAH and are already on stable treatment, you may be eligible to join the IMPROVE-PAH trial and potentially access IKT-001 before it becomes widely available.
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.
Clinical trialRSS2 days ago
The FDA has approved a clinical trial for a new cell therapy called remestemcel-L-rknd made by Mesoblast. The therapy will be tested in children ages 5-9 who have Duchenne muscular dystrophy (DMD), a serious muscle disease. About 76 children will participate in the trial and receive either the therapy or a placebo while continuing their regular DMD treatments.
WHY IT MATTERSThis trial represents a new treatment approach for DMD in young children who are still in a critical window for muscle development, offering families a potential alternative or complement to existing therapies.
Clinical trialCLINICALTRIALSApr 17
Researchers are testing a new gene therapy called BBP-812 to treat Canavan disease, a rare brain disorder that affects children. The therapy uses a modified virus to deliver a healthy copy of a gene that's missing or broken in people with this disease. This early-stage trial will check if the treatment is safe and whether it helps patients.
WHY IT MATTERSThis trial is now actively recruiting children with Canavan disease — if your child has been diagnosed, you may be eligible to participate in one of the first human tests of this gene therapy approach.
Clinical trialUNITERAREApr 3
This is a clinical trial testing a blood clotting medicine called Nuwiq in women and girls with hemophilia A who need surgery. Hemophilia A is a rare bleeding disorder where the body doesn't make enough of a protein that helps blood clot. The trial will study how well Nuwiq works and what dose is best for these patients during surgical procedures.
WHY IT MATTERSThis trial is specifically designed for women and girls with hemophilia A undergoing surgery — a population that often receives less research attention than males with the same condition.
Clinical trialUNITERAREApr 3
Triple Hair Inc is recruiting patients for a Phase 3 clinical trial testing a new treatment called TH07 for androgenic alopecia, commonly known as male or female pattern baldness. This is a large-scale study to confirm whether TH07 works better than current treatments. If you have pattern hair loss, you may be eligible to join and help test this potential new therapy.
WHY IT MATTERSThis Phase 3 trial is actively recruiting now, meaning if you have androgenic alopecia, you could potentially enroll and access an investigational treatment while contributing to evidence that could lead to FDA approval.