Measuring the Efficacy of Tafamidis as a Treatment for ATTR-CM
source: pixabay.com

Measuring the Efficacy of Tafamidis as a Treatment for ATTR-CM

When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…

Continue Reading Measuring the Efficacy of Tafamidis as a Treatment for ATTR-CM
NY Giants Running Back Saquon Barkley Spotlights Rare Disease 22q on Good Morning America
Source: Pixabay.com

NY Giants Running Back Saquon Barkley Spotlights Rare Disease 22q on Good Morning America

As part of the NFL's "My Cause, My Cleats" awareness project, New York Giants Running back Saquon Barkley chose 22q11.2 deletion syndrome (22q) as his cause to highlight! And while that…

Continue Reading NY Giants Running Back Saquon Barkley Spotlights Rare Disease 22q on Good Morning America
Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome
Source: Pixabay

Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome

According to a story from Newswise, the Seaver Autism Center for Research and Treatment at Mount Sinai recently announced that patient enrollment has begun for a clinical trial that will…

Continue Reading Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome