Other examples include obesity, Gaucher's disease, phenylketonuria (PKU), medium-chain acyl-CoA dehydrogenase deficiency (MCADD), maple syrup urine disease (MSUD), and hemochromatosis. These ...
The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the <a ta ...
Single gene disorders, like Huntington’s disease and cystic fibrosis, actually follow Mendelian inheritance patterns. The PKU-associated enzyme deficiency was determined biochemically in the ...
GENEVA, SWITZARLAND / ACCESSWIRE / October 29, 2024 /RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief, or the Company), a biopharmaceutical company committed to delivering ...
Q3 2024 Earnings Call Transcript November 7, 2024 PTC Therapeutics, Inc. beats earnings expectations. Reported EPS is $-1.39, ...
The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback