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The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...
I care deeply because this chronic disease is mine. It lives inside me. It’s radically changed my life and the life of my ...
Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...
Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~~ ...
Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines ...
The FDA has approved Cabometyx for some patients 12 years old and older with previously treated neuroendocrine tumors.
The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...
Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...
Industry leaders gather to discuss innovative solutions for accelerating the development of therapeuticsBOSTON, MA, April 10, ...
while a Phase IIa study demonstrated reduced disease flares and steroid use over a 52-week treatment period in patients with IgG4-RD. 1 “Orphan drug designation for these two rare, immune-mediated ...
ATSN-201 was given regenerative medicine advanced therapy designation to treat X-linked retinoschisis, for which there ...