News

Horizon Pharma Support for People Impacted by Hurricane Harvey

At Horizon Pharma, our thoughts continue to be with the people of southeast Texas who are facing devastation caused by Hurricane Harvey. While we work with partners to provide support for the Texas community, we want to ensure that those impacted who are living with urea cycle disorders (UCDs), chronic…

Mount Kilimanjaro Climb to Raise Money for the Cystinosis Research Foundation

Mount Kilimanjaro Climb by RuthAnn Ahnen in honor of her daughter Katie Ahnen RuthAnn Ahnen began her journey on December 30, 2014 to climb Mount Kilimanjaro to raise money for the Cystinosis Research Foundation in honor of her daughter Katie and the cystinosis community. (Pictured are Katie and her friend…

AFTER YEARS OF WAITING, WE HAVE RECEIVED FDA APPROVAL FOR CYSTEAMINE TODAY!

The drug developed by Raptor Pharmaceuticals will be sold under the name Procysbi. View the Article from the New York Times Read coverage from MarketWatch.com Read the Press Release from Raptor Pharmaceuticals Watch more videos on Raptor Pharmaceutical Corporation’s Cystinosis Treatments

SIGMA-TAU PHARMACEUTICALS LAUNCHES CYSTARAN™FOR THE TREATMENT OF CORNEAL CYSTINE CRYSTAL ACCUMULATION

GAITHERSBURG, MD, May 1, 2013—Sigma-Tau Pharmaceuticals, Inc. (Sigma-Tau), a part of the Sigma-Tau Group Rare Disease Franchise, announced today the availability of CYSTARAN™ (cysteamine ophthalmic solution) 0.44%, the first and only FDA-approved therapy for the treatment of corneal cystine crystal accumulation in patients with cystinosis. Sigma-Tau developed CYSTARAN in partnership with…

CRF Raises $2.2 Million To Cure Cystinosis!

Researchers Gain In Developing New Treatments For Rare Disease NEWPORT BEACH, Calif. — The Cystinosis Research Foundation raised $2.2 million at its 11th anniversary celebration of Natalie’s Wish, where it was announced that researchers believe a promising new treatment to save the eyesight of cystinosis patients may be ready for…