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

In a touching tale of humanitarianism, Selva, an 11-year-old boy from Tiruchirappalli who survived Fanconi Anemia, met the ...

Canaccord Genuity analyst Whitney Ijem maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report) yesterday and set a ...

New Delhi: The Central government is set to launch a scheme to develop 12 indigenous drugs for eight rare diseases, health ...

The Lagos University Teaching Hospital (LUTH), in collaboration with the Sickle Cell Foundation Nigeria (SCFN), has launched a bone marrow transplant program to cure sickle cell disease in Nigeria. In ...

Anemia is highly prevalent in patients with chronic heart failure (HF) and is associated with poor clinical outcomes. Multiple mechanisms contribute to anemia in chronic HF, and subnormal ...

(NASDAQ:RCKT) announced that it had been able to complete enrollment for its pivotal phase 2 study using its gene therapy RP-A501 for the treatment of male patients with Danon disease. The ...

It has long been known that when red blood cells break down or anemia occurs due to bleeding ... Analyzing the lipids and related proteins in the bone marrow of anemic mice showed that although ...

Rocket’s lentiviral vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and ...

Given the prevalence of Danon disease across regions, the Company plans to pursue regulatory filings concomitantly in the U.S. and ex-U.S. "From a clinical perspective, the important thing is that ...

Her doctors performed genetic tests and discovered she had a rare blood disorder, called Fanconi’s Anemia, that would require a bone marrow transplant, among other treatments ... received chemotherapy ...