Pompe Disease

Jan 05, 2022

Although COVID-19 continued to dominate headlines in 2021, pharmaceutical companies did not cease developing new treatments this year. The US Food and Drug Administration maintained a rapid rate of new drug approvals this year, all while managing the urgent examination of COVID-19 tests, treatments, and vaccines un...

May 07, 2020

Avrobio taps ADC of Magenta for enhancing gene therapy conditioning Avrobio is working to make conditioning safer. The company is collaborating with Magenta Therapeutics to check antibody-drug conjugate. Under the agreement, the partners will evaluate Magenta’s lead conditioning program, MGTA-117, with at lea...

Oct 13, 2015

Introduction: Any disease that affects less than 200,000 patients or 1 in 1500 is termed as a rare disease. There are around 5000 to 8000 rare disease reported, out which only 10% have cure. According to the European Organization for rare disease, 80% of rare diseases are genetic in nature. Some of the rare disease...