David Fajgenbaum’s life changed forever one summer day in 2010. After experiencing fatigue, his liver, kidneys, bone marrow, heart, and lungs suddenly began to shut down. He was admitted to the intensive care unit where doctors found that he had a retinal hemorrhage that left him blind in his left eye. David drifted in and out of consciousness, was put on a feeding tube, and received blood transfusions. In weeks, the 26-year-old medical student sharply deteriorated and was given last rites.

The U.S. Food and Drug Administration approved Clinuvel Pharmaceuticals Ltd.’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending the Australian drugmaker’s shares to a record high.

Deep Genomics’ proprietary artificial intelligence-based drug discovery platform identified a novel treatment target and corresponding drug candidate for the rare and potentially life-threatening genetic disorder Wilson disease.

Genentech released full data from the company’s pivotal Phase III SAkuraStar trial of satralizumab as a monotherapy for NMOSD.

Sarepta Therapeutics Inc. was informed that an adverse event report was erroneously submitted to the U.S. health regulator regarding an ongoing study of the company’s gene therapy for Duchenne muscular dystrophy (DMD).

The U.S. Food and Drug Administration approved Daiichi Sankyo Co. Ltd.’s Turalio for adult patients with a type of rare, non-cancerous tumor affecting joints and limbs.

The U.S. FDA gave Alexion Pharmaceuticals a thumbs-up for Soliris (eculizumab) to treat neuromyelitis optica spectrum disorder in adults that express a specific biomarker.

Mallinckrodt Plc tentatively agreed to pay $15.4 million to resolve a U.S. Justice Department probe into how a drugmaker that the company now owns marketed an expensive treatment for a rare infant seizure disorder and multiple sclerosis.

With the books closed on another ASCO meeting, it’s clear to see that checkpoint inhibitors keep showing a promise.

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.