Top 50 Thought-Leaders in Orphan Drugs and Rare Disease: New Report


This report, created by Terrapinn and the World Orphan Drug Conference recognizes the top 50 thought-leaders in orphan drugs and rare disease. Chris Austin, Director, National Center for Advancing Translational Sciences, NIH In September 2012, Christopher P. Austin, M.D., was appointed the first permanent director of […]

NYSCF Partners NIH to Generate Stem Cell for Treating Rare Diseases


In what is anticipated to be a major step forward for rare disease research, The New York Stem Cell Foundation (NYSCF) Research Institute is partnering with the National Institutes of Health (NIH) Undiagnosed Disease Program (UDP). NYSCF scientists will generate stem cell lines from 100 patients in […]

Questions on Caregiving? Ask the Experts Today!


Global Genes  | RARE Project will be hosting a webinar this week on November 21st at 11:00AM PT (90M) and we’d like to invite YOU to participate in the conversation. The topic of the webinar is on caregiving: “Taking Care of Yourself While Taking Care of Your Loved […]

FDA Sets Up New Rare Disease Research Fund


The US Food and Drug Administration (FDA) is to support the research of treatment for rare diseases through a new $14m fund. The money will support 15 research projects that are investigating new ways to tackle rare diseases, defined in the US as conditions that affect […]

The Global Genes | Rare Project Named Top-Rated NonProfit 2013 by GreatNonProfits


GreatNonprofits is the leading developer of tools that allow people to find, review and share information about great– and perhaps not yet great– nonprofits and charities. Thanks to the reviews of the rare disease community, Global Genes was named a Top-Rated NonProfit for 2013 by […]

Chiesi Group Acquires Zymenex, Enters Rare Disease Market


Chiesi Group will acquire Zymenex Holding and its related companies from its venture capital investor-owner Sunstone Capital for an undisclosed price, in a deal that marks Chiesi’s entrance into the rare disease space and, according to the company, provides an opportunity to access high-potential markets. Founded in […]

Rep. Herrera Beutler’s Baby Defies Odds of Usually-Fatal Condition of Potter’s Syndrome

Potter's Syndrome

Before Rep. Jaime Herrera Beutler’s baby was even born, the odds seemed against her surviving. She hadn’t developed kidneys, which meant she didn’t produce urine which is the main ingredient of amniotic fluid. Without amniotic fluid, a baby’s lungs can’t develop properly. But today, little […]