Pyruvate kinase deficiency (PKD) is an autosomal recessive genetic disease caused by mutations in the PKLR gene. To date, the clinical manifestations of PKD are heterogeneous, ranging from fetal ...

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable ...

The fall in AGIO stock comes after reports of an unexpected side effect linked to its sole-marketed drug Pyrukynd in thalassemia patients.

Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, announced today the ...

The Phase 3 ENERGIZE and ENERGIZE-T trials enrolled a total of 452 patients reflective of the real-world thalassemia population. The results demonstrated that mitapivat improves hemolytic anemia and ...

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They ...

Lindy Noel's mom always knew her daughter was going to change the world. She just didn't know it would be the world of NHL ...

Proliferating cells, in particular tumor cells, express a dimeric isoenzyme of pyruvate kinase, termed Tumor M2 pyruvate kinase. In the last few years, much attention has been paid to this novel ...

Zurcher Kantonalbank Zurich Cantonalbank increased its position in Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT – Free Report) ...

Despite a delay in the RP-L301’s pivotal study for Pyruvate Kinase Deficiency (PKD), these developments collectively reduce the risks associated with the ongoing pivotal DD study, justifying the ...