Proteostasis deficiencies

This early-stage study tested a new drug called SAR445514, which helps the body's natural killer (NK) cells attack cancer cells in people with multiple myeloma or light-chain amyloidosis that had returned or stopped responding to other treatments. The study was terminated early, …

This study tested a drug called inotersen in 31 people with a rare condition where a faulty protein builds up in the heart, causing damage. The drug is designed to lower levels of that protein. The trial was stopped early, so it's unclear if it works, but researchers hoped to see…

This phase 3 study tested an experimental drug called birtamimab in 208 people with advanced AL amyloidosis, a rare disease where abnormal proteins damage organs like the heart. Participants received either birtamimab or a placebo, plus standard chemotherapy. The goal was to see …

This study tested a drug called ixazomib plus dexamethasone against other standard treatments for people with relapsed AL amyloidosis, a rare disease where abnormal proteins build up in organs. The trial aimed to see if the combination improved blood markers and slowed heart or k…

This study looked at whether a drug called K0706 is safe and tolerable for people with Dementia with Lewy Bodies (DLB). Researchers planned to give 45 participants either a low dose, high dose, or placebo for 12 weeks, then check for side effects and measure changes in brain and …

This study tested a drug called AVP-786 to see if it could safely reduce disinhibition—impulsive or inappropriate behavior—in people with neurodegenerative disorders like Alzheimer's or frontotemporal dementia. The trial planned to include many participants but was terminated ear…

This study tested a personalized touchscreen device (My PATI) to help people with Alzheimer's or related dementias communicate their care preferences. The goal was to improve quality of life for both patients and their caregivers. The study was terminated early, so results are li…

This study tested a drug called CVL-871 to see if it is safe and can help reduce apathy (lack of motivation or interest) in people with dementia. The trial included 41 participants with mild to moderate dementia and clinically significant apathy. The main goal was to check for si…

This early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.

This study looked at why people with type 1 diabetes often fail to sense dangerously low blood sugar (hypoglycemia). Researchers tested how opioid drugs like morphine and naloxone affect the body's ability to respond to low sugar. The goal was to better understand the problem, no…

This study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…