Dancing Through Diagnosis: Alnylam’s Givlaari Campaign Puts Rare Disease in the Spotlight

Alnylam Pharmaceuticals has launched a unique promotional campaign for Givlaari, its treatment for acute hepatic porphyria (AHP), that swaps the usual clinical narratives for something more artistic: dance. As reported…

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World’s First Personalized Gene-Editing Therapy Offers Hope to Infant with Rare Disease

In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…

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Resuming VYKAT™ XR Treatment Shows Significant Benefits for Prader-Willi Syndrome Patients, New Data Reveals

Soleno Therapeutics recently announced new findings regarding its investigational drug VYKAT™ XR (diazoxide choline extended-release tablets) for the treatment of Prader-Willi syndrome (PWS), as detailed at GlobeNewswire. The data, which…

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