The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial. Shortly after the regulatory hold was implemented, Intellia disclosed that ...

Intrapleural enzyme therapy (IET) improves outcomes in adult inpatients with complicated parapneumonic pleural effusion or empyema.

Regenxbio Inc. faces pivotal catalysts in 2026, notably RGX-121's FDA decision and RGX-202 Duchenne data, potentially ...

Sharp Therapeutics Corp. ("Sharp" or the "Company") (TSX-V: SHRX) (OTCQB: SHRXF), a biotechnology company developing small-molecule therapeutics for genetic diseases, announced today the appointment ...

Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

A new study published in the Chest Journal showed that Full MIST2 dosing of intrapleural enzyme therapy (IET) was Linked to Better Outcomes but infrequently administered and showed ...

Tirzepatide users on hormone therapy had added improvements in weight, cardiometabolic mar ...

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...

Chronic pain affects more than 50 million Americans, yet for decades, treatment options for pain that persists in the absence ...

Unique patent policy, co-funding, and industry mentorship help move discoveries at Rutgers across the “valley of death.” ...

ClearPoint Neuro is positioned for robust growth in 2026, driven by IRRAS, the PRISM 1.5T system clearance, and the company's new CRO facility. While the immediate future of uniQure's AMT-130 remains ...