Medicines in development for rare diseases
17 June 2016

Rare diseases, when taken together, are not that rare at all.

Body’s RNA-editing system targeted to treat drug-resistant leukemia
15 June 2016

University of California, San Diego researchers have identified the process by which the precancerous precursors of white blood cells become leukemia stem cells: the human body's own gene-editing p

Ohio scientists block chromatin-modifying enzyme, suppressing glioblastoma growth
15 June 2016

Scientists from Ohio State University Comprehensive Cancer Center have identified a new player in the growth of an aggressive brain tumor, or glioblastoma.

AbbVie and CytomX Announce Strategic Collaboration for Probody Drug Conjugates
15 June 2016

AbbVie (ABBV)  and CytomX Therapeutics, Inc.

CF Patients Will Likely Benefit from Promising, New Data on Two Drugs
15 June 2016

Well known pharmaceutical company recently revealed new clinical data on real-world long-term outcomes in cystic fibrosis (CF) patients treated with ivacaftor.

FDA Approves Higher Dose of Orfadin for HT-1
15 June 2016

The FDA is busy today. Earlier today, we reported on the FDA approving an at-home infusion option of Glassia for patients with  AAT-deficiency.

FDA Approves At-home Treatment for AAT Deficiency
15 June 2016

The FDA has approved an expanded label for Glassia [Alpha-1 Proteinase Inhibitor (Human)], so that adult patients with emphysema due to severe alpha-1 antitrypsin (AAT) deficiency can now self-infu

Quality of Life, Nivolumab, and Advanced Renal Cell Carcinoma
14 June 2016

An analysis just published in The Lancet demonstrates higher quality of life in patients with advanced renal cell carcinoma taking nivolumab compared to everolimus.