Aardvark Therapeutics has raised $29 million in financing to help support three planned Phase 2 clinical trials of its…
Articles by Marisa Wexler, MS
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
People with Prader-Willi syndrome (PWS) and other diseases commonly experience sleep problems, and the problems are rarely addressed effectively…
Specific genetic subtypes and growth hormone use both influence the physical characteristics, such as head shape and eye appearance, of…
Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases,…
Tonix Pharmaceuticals has licensed technology from the French National Institute of Health and Medical Research (Inserm) that it…
Inversago Pharma‘s INV-101 has been granted rare pediatric disease (RPD) designation by the U.S. Food and Drug Administration for…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Measuring how electricity moves through the body could help detect low-grade inflammation in adults with Prader-Willi syndrome (PWS) without…