by Peter Ciszewski | May 29, 2019
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA), a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye...
by Peter Ciszewski | May 29, 2019
The US Food and Drug Administration granted orphan drug designation for AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in the late stages of preclinical development for treating children with galactosemia. Galactosemia, a rare...
by Peter Ciszewski | May 28, 2019
Kristen Wheeden, Director of Development for the American Porphyria Foundation, discusses current treatment options for Porphyria, including some treatments in clinical trials. Porphyria is caused by different inherited disorders in the synthesis of heme, the...
by Peter Ciszewski | May 27, 2019
Prof. Arndt Rolfs, MD, Chief Executive Officer at Centogene discuses the challenges of diagnosing rare diseases. Physicians learn early in their careers that making a proper diagnosis is the first step in ensuring that patients are properly treated for their...
by Peter Ciszewski | May 22, 2019
The US Food and Drug Administration (FDA) has cleared Abeona Therapeutics to begin a Phase 1/2 clinical trial evaluating its novel, one-time gene therapy ABO-202 for the treatment of CLN1 disease. ABO-202 is designed to deliver a functional copy of the PPT1 gene to...
