CHICAGO – Scientists at Stanford University School of Medicine have used the CRISPR gene editing tool to repair the gene that causes sickle cell disease in stem cells from diseased patients, paving the way for a potential cure for the disease, which affects up to 5 million people globally. “What we’ve finally shown is that […]
ORDIndia announces research collaboration with George Mason University, USA with the newly established center for the study of chronic metabolic and rare diseases (CSCMRD). ORDI co-founder, Dr. Harsha Rajasimha to serve as Co-Director of the center along with Dr. Ancha Baranova, Associate Professor, George Mason University. The center webpages can be accessed at: http://cscmd.cos.gmu.edu/index.html Rare […]
Prajwal Hulluraiah is engrossed in a game on his mother’s mobile phone and wouldn’t look up to talk to you. But that’s where his similarity with any other 11-year-old ends. Twice a month, Prajwal checks into the Indira Gandhi Institute of Child Health (IGICH) in Bangalore for a six-hour procedure to replace the enzymes in […]
According to sources, the cost of treatment would run into thirty-five crore rupees annually, which will be footed by the State Government. Bengaluru: Parents and guardians of patients suffering from rare diseases have always found it difficult to arrange funds for the treatment of their children with rare diseases. However, with the Karnataka high court […]
Developing medicines for rare diseases requires an entirely different mindset, says John Crowley, chairman and CEO of rare disease company Amicus Therapeutics. “Developing drugs in rare diseases is very different from more prevalent disorders – the clinical studies are very small, you have different endpoints and biomarkers, different regulatory paradigms and different types of sales […]
Kochi, Kerala, India.