From respiratory infection to fatal neurological disease, EHV-1 demands rapid PCR diagnosis, and detection protocols impact ...
So in general, really blinatumomab was very well tolerated. There were some more cytopenias in some of the patients with ...
Biotinidase deficiency (BTD) is an inherited metabolic disorder with neurological, dermatological and metabolic manifestations. Clinical deterioration in adequately treated older children is uncommon ...
The US FDA approved 46 new drugs in 2025, despite a tumultuous year at the regulatory agency.
Biohaven's troriluzole candidate for spinocerebellar ataxia (SCA) has been turned down by the FDA, causing shares in the company to lose more than 40% of their value in pre-market trading. The news is ...
After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU. Skyclarys ...
Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ataxia, is missing. Experiments in worms, human cells, and mice revealed ...
This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...
Abstract: Cerebellar ataxia is the poorly coordinated movement that results from injury or disease affecting the cerebellum. The diagnosis and assessment of ataxia are significantly challenging due to ...
AML develops quickly, with symptoms like bone pain and shortness of breath, while CML progresses slowly and can start with weight loss and night sweats. AML is more common in both children and adults, ...