Boston-based Alexion Pharmaceuticals announced positive topline data from the company’s Phase III trial of Ultomiris (ravulizumab-cwvz) in adults with generalized myasthenia gravis.

A study found potential in a novel gene therapy method to help children born with a rare genetic and neurodegenerative disorder, AADC deficiency.

The U.S. Food and Drug Administration is planning a new warning for Johnson & Johnson’s Covid-19 vaccine over a rare but serious side effect, Guillain-Barré syndrome (GBS).

The IPO train continues to barrel down the tracks as more life sciences companies announced and closed their initial public offerings, bringing millions more to their coffers. 

Intellia Therapeutics and Regeneron Pharmaceuticals shook the biotech and CRISPR world on June 26, announcing positive early data from the first-ever patients to have their DNA edited with an in vivo CRISPR/Cas9 therapy delivered systemically.

The European Commission approved Roche’s injectible drug Enspryng, which can be administered at home to treat neuromyelitis optica spectrum disorder.

Shares of Vertex Pharmaceuticals fell in trading after the company announced it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study for a rare disease that can lead to complications of the liver and lungs.

It’s hard to address something that you cannot define. Rare disease management is a high priority for payers. In a recent rare disease trend report, 90% of surveyed payer respondents ranked rare disease management as a high or moderate priority. This is due primarily to the significant budget impact of therapies that may be in the hundreds of thousands of dollars

The U.S. Food and Drug Administration granted marketing clearance for Ryplazim as the first FDA-approved therapy for the treatment of patients with the plasminogen deficiency type 1 (hypoplasminogenia), a rare genetic disorder.

Several biopharmaceutical companies submitted New Drug Applications (NDAs) to the U.S. Food and Drug Administration in recent weeks, covering treatment indications ranging from an opioid overdose to bipolar disorder and rare disease.