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This strategic financing allows Cycle to build on the success of NITYR (nitisinone) Tablets, which treats patients with the rare genetic metabolic disease, hereditary tyrosinemia type 1. Cycle’s

The German company said that 41% of PAH patients transitioning to Adempas therapy after inadequate response to  phosphodiesterase-5 inhibitors (PDE5 inhibitors) achieved the composite primary endpoint, which is

The term “orphan drugs” refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the United States, an orphan disease is

Ibrutinib, a once-a-day drug that is administered orally, inhibits the first-in-class Bruton’s tyrosine kinase (BTK) that sends crucial signals to B cells to mature and produce antibodies, said

The post hoc analysis from the pivotal Phase III IRIDIUM study—presented virtually at the European Respiratory Society (ERS) International Congress 2020— also showed the safety profile for high-dose

Alkahest is a clinical stage company that is focussed on the discovery and development of treatments for neurodegenerative and age-related diseases with therapies that target the aging plasma

Enrollment has been completed for the AmpionTM Phase I US based clinical trial for IV treatment of COVID-19 patients requiring supplemental oxygen. The final results from the 5-day

Gavreto, a once-daily and oral precision therapy, will help selectively target RET alterations such as fusions and mutations irrespective of the tissue of origin. Roche’s wholly-owned member Genentech

PANS is a condition defined by sudden onset of obsessive-compulsive symptoms and/or severe eating restrictions, along with at least two other cognitive, behavioral, or neurological symptoms, according to