As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in ...

The woody breast (WB) myopathy poses significant economic and welfare concerns to the poultry industry, however, there is no effective strategy to mitigate this pathology due to its unknown etiology.

Sep. 9, 2024 — Scientists developed a highly promising gene therapy to treat glaucoma -- a debilitating eye condition that can lead to complete vision loss, and which affects around 80 million ...

Nat Clin Pract Neurol. 2006;2(9):482-493.

including cases of liver toxicity and deaths in a trial of its AT132 gene therapy candidate for rare disease X-linked myotubular myopathy (XLMTM) which resulted in a clinical hold in 2021.