New treatments and completed trials across every condition — what was tested, what was found, and what it means for patients.
Condition guides
Anxiety disorders affect about 30% of adults at some point in life.
Read the guide →Acute coronary syndrome includes heart attacks and unstable angina — conditions where blood flow to the heart muscle is suddenly reduced or blocked.
Read the guide →Acute kidney injury happens when the kidneys suddenly stop filtering waste from the blood, often during hospitalization for severe illness, surgery, or sepsis.
Read the guide →Atherosclerosis is the buildup of cholesterol plaques in artery walls, underlying most heart attacks and strokes.
Read the guide →Cardiovascular disease remains the leading cause of death worldwide, encompassing conditions like coronary artery disease, heart failure, arrhythmias, and stroke.
Read the guide →Cardiovascular disease remains the leading cause of death worldwide, but treatment has advanced dramatically.
Read the guide →Researchers tested a one-time gene transfer therapy given through the spine in children with spinal muscular atrophy who could sit but couldn't walk. After one year, children who received the treatment showed significantly better muscle function compared to those who didn't.
What this means for patients
This one-time gene therapy is FDA-approved for spinal muscular atrophy under the brand name Zolgensma, though this specific delivery method (through the spine) may have different approval status. If your child has spinal muscular atrophy, talk to your neuromuscular specialist about whether this treatment approach is available and appropriate for your child's specific situation.
The RUBY trial tested reni-cel, a one-time gene editing treatment for patients with severe sickle cell disease who experience frequent painful crises. Researchers modified each patient's own stem cells to reactivate fetal hemoglobin — preventing cells from taking the sickle shape that causes blockages and organ damage.
Read →TEMPO-3 tested tavapadon — a once-daily selective dopamine pill — added to levodopa in 507 people with Parkinson's and motor fluctuations. Tavapadon added about 1.1 hours of good on-time per day.
Read →OCULUS tested whether holding one weekly or daily dose of a GLP-1 drug (like Ozempic or Mounjaro) before upper endoscopy reduces stomach contents that complicate sedation. The trial was stopped early — 25% who continued had retained food vs. 3% who held the dose.
Read →A daily targeted pill reduced disease progression risk by 84% in unresectable Stage III EGFR-mutated non-small cell lung cancer after chemoradiation — a population with few good options after completing standard treatment.
Read →STRENGTH tested intrathecal onasemnogene abeparvovec — a spinal-fluid version of Zolgensma — in 27 children and teens with SMA who had stopped nusinersen or risdiplam. The 52-week safety profile matched what was seen in younger, untreated patients.
Read →Adding abemaciclib to hormone therapy after surgery cut the risk of cancer returning by 35% in high-risk hormone receptor-positive breast cancer patients — a meaningful improvement over hormone therapy alone.
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