The drug Vyondys 53 has met a surrogate endpoint of protein production, but there’s no clear proof it has a clinical benefit.

The company and its partner halted two clinical trials for futility early this year, but now say they’ll seek approval for aducanumab.

A look at some of the circulating molecules that may indicate various Alzheimer’s pathologies and serve as the bases of noninvasive tests for the disease.

Researchers hope circulating biomarkers will enable earlier detection and better monitoring of the neurodegenerative disorder—and perhaps help usher in new treatments.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

Several early-stage clinical trials indicate that implanting patients with the cells is safe. But whether they can alleviate neurological problems remains to be seen.

The move will allow the Belgium-headquartered biopharmaceutical giant to boost its work on treatments for rare diseases.

The widely expected move comes months after the companies scrapped trials of another Alzheimer’s drug.