Belite Bio reports landmark phase 3 success for Stargardt Disease therapy

Seeing well-controlled Phase 3 data that shows a marked slowing of lesion growth in Stargardt disease is deeply encouraging

Valneva Phase 2 data for Lyme disease vaccine candidate shows striking immune response

Pfizer and Valneva have been collaborating on VLA15’s development and commercialization since April 2020

Teva breaks ground with EU approval for two key bone disease biosimilars

Siolta Therapeutics’ novel microbiome therapy shows promise in preventing allergic diseases in infants

Cardiol Therapeutics wins US patent protection for heart-disease drug platform

The patent will lock in broad protection for the company’s lead therapy CardiolRx and its novel subcutaneous candidate CRD-38

Chiesi inks agreement with AbbVie subsidiary to push next-generation brain-targeting therapies for rare diseases

Kaiser Permanente’s study highlights power of genetics in preventing heart disease

Cardiovascular disease (CVD) is the leading cause of death in the US and worldwide with over 82 million Americans living with CVD

Biohaven disappointed as rare disease therapy faces FDA setback 'despite promising results'

The FDA setback highlights ongoing tensions between patient needs, regulatory processes, and the use of real-world evidence in evaluating treatments for rare diseases

FDA approves UCB’s Kygevvi for rare genetic mitochondrial disease TK2 deficiency

Kygevvi combines two pyrimidine nucleosides, doxecitine and doxribtimine, which act by integrating deoxycytidine and deoxythymidine into skeletal muscle mitochondrial DNA

Quest Diagnostics’ study confirms accuracy of blood tests for Alzheimer’s disease diagnosis

Quest Diagnostics’ blood-based test panels demonstrate confirmatory accuracy, potentially reducing the need for PET imaging