Rare blood diseases – PharmaLive

Investors Hand Disc Medicine $90 Million to Control Hematological Diseases

Biopharma, Business, Erythropoietic Protoporphyria (EPP), Financing, Hematologic Diseases, Investors, Metabolism, Myelofibrosis, R&D, Rare blood diseases, Red Blood Cells, Series B

Investors Hand Disc Medicine $90 Million to Control Hematological Diseases Published: Sept. 2, 2021 By Heather McKenzie BioSpace A two-year-old biopharma company tackling rare blood diseases has received a […]

September 2, 2021/by Andrew Humphreys

FDA Approves First Drug to Treat Group of Rare Blood Disorders in Nearly 14 Years

Approvals, Eosinophils, FDA, FDA/Regulatory, Hypereosinophilic Syndrome (HES), Organ Damage, Rare blood diseases, White Blood Cells

The U.S. Food and Drug Administration approved Nucala (mepolizumab) for adults and children aged 12 years and older with hypereosinophilic syndrome (HES) for six months or longer without another identifiable non-blood related cause of the disease.

September 25, 2020/by Andrew Humphreys

Apellis’ PNH Drug Beats Alexion’s Soliris in Phase III

Blockbusters, Blood Diseases, Clinical Trial Endpoints, Clinical Trials, Hemoglobin levels, Paroxysmal Nocturnal Hemoglobinuria (PNH), R&D, Rare blood diseases, Red Blood Cells, Stocks, Superiority

Apellis Pharmaceuticals’ pegcetacoplan beat out Alexion Pharmaceuticals’ Soliris in the Phase III PEGASUS study in adults with paroxysmal nocturnal hemoglobinuria (PNH).

January 7, 2020/by BioSpace

Japan approves test of iPS cells for spinal injuries

Clinical Trials, Embryonic stem cells, Health ministry panel, Human Induced Pluripotent Stem Cells, Japan, Japanese Ministry of Health, Mature cells, R&D, Rare blood diseases, Rare eye diseases, Reprogrammed stem cells, Scientists, Skin

Japanese scientists will test the use of human-induced pluripotent stem cells (iPS) to treat spinal cord injuries, a health ministry panel that approved the research project said.

February 18, 2019/by Reuters Health