The Phase Ib trial is set to test a gene therapy designed to target a rare disorder that causes muscle and respiratory failure.

Xenon gas, currently used in medicine as an anesthetic and neuroprotective agent for treating brain injuries, showed potential in protecting the brain in studies.

The Covid-19 outbreak in 2020 was on a scale not seen since the Spanish flu pandemic in 1918 and laid bare our lack of ...

Autoimmune diseases represent a significant challenge in the field of medicine, as they occur when the immune system erroneously targets the body's own ...

Delve into the increasingly dynamic landscape of ALS treatment development with thirteen companies advancing candidates in ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

A novel wireless photothermal DBS nanosystem uses nanoparticles to target and restore damaged neurons in Parkinson’s disease, ...

FAPI PET—can detect the first signs of tissue remodeling in patients with pulmonary arterial hypertension (PAH), providing physicians with an early marker for disease progression. This approach has ...

Trypanosomes are parasites that cause sleeping sickness, Chagas disease, and various animal diseases. Diagnosis and treatment remain complex and no effective vaccine has been developed. A better ...

Study highlights use of techniques such as long-read genome sequencing, optical genome mapping, and RNA sequencing for rare ...

Below is a review of novel drug and biological products approved by the Food and Drug Administration (FDA) in 2024. The list includes several groundbreaking therapies that are expected to change the ...

MRI of inhaled perfluoropropane can assess regional lung ventilation and monitor improvements in response to medication ...