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.
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.
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.
ResearchRSS2 days ago
Researchers found that a Parkinson's disease medication called Crexont helped patients spend more time feeling better and less time experiencing symptoms when they switched from other levodopa treatments. The study showed improvements in movement control and daily functioning across different patient groups. This is early-stage research being presented at a medical conference.
WHY IT MATTERSIf you take levodopa for Parkinson's and struggle with 'off' periods when symptoms return between doses, Crexont may offer a way to extend your good symptom control time throughout the day.
ResearchRSS2 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.
ResearchPUBMED3 days ago
Linear interstitial keratitis (LIK) is a very rare eye condition where a thin line of cloudiness forms in the clear part of the eye (the cornea). This study looked at 6 patients with this condition to better understand what it looks like, how doctors can diagnose it, and what treatments work best. The researchers found that the cloudy line usually appears near the edge of the cornea and can be seen clearly with special eye imaging tools.
WHY IT MATTERSThis is the largest study to date on linear interstitial keratitis, providing eye doctors with new guidance on diagnosis and treatment options for a condition that previously had no agreed-upon management approach.