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 ...

Fanconi anemia (FA) is a rare genetic disease, affecting 1 in 160,000 people. The condition leads to gradual bone marrow failure in about 90% of people affected. Bone marrow is the spongy material ...

(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 ...

Bone marrow is a spongy organ that fills the center of various bones of your body. It is where stem cells produce red and white blood cells and platelets. Without bone marrow, you couldn't move oxygen ...

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 ...

Bone marrow transplant for aplastic anemia Aplastic anemia is a rare disease that causes your bone marrow to stop producing blood cells, making it a very serious illness. A blood transfusion can ...

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 ...