Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
To know Bentley Shelton is to love him. “He brings light to everybody that meets him,” said Brittany Shelton, Bentley’s mom. “He always ...
GlobalData on MSN15h
Travere takes rare disease kidney drug to FDA despite failed trialTravere’s stock price climbed by 14% following the announcement that it will seek an FDA label expansion for Filspari.
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
Rare diseases have become an attractive target for pharmaceutical companies both on the ASX and internationally.
A surprising new study suggests that COVID-19 may not have originated from bats or pangolins, but rather from a rare fusion of human diseases. Using an advanced AI-driven approach called max-logistic ...
TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...
Mereo BioPharma shifts focus to rare diseases with setrusumab showing strong results for Osteogenesis Imperfecta. Learn more on MREO stock here.
Background SLE is a systemic autoimmune disease with a large number of common risk gene variants, but several rare gene ...
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Barcelona-based sales agency Jambika Docs has acquired the documentary feature “Noah” ahead of the European Film Market in ...
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