by CheckRare Staff | May 4, 2023
Aimee Donald, MBChB, PhD, Pediatrician at Royal Manchester Children’s Hospital, and Professor at the University of Manchester, discusses the PROCEED and PROVIDE gene therapy trials testing PR001 in Gaucher disease patients. Gaucher disease arises from...
by CheckRare Staff | Apr 25, 2023
« Prev1 / 1Next »CTCL Challenges Managing Minority Populations (Full interview)CTCL: Challenges of Treating People of Color (Chapter 1)CTCL: Financial Barriers in the Latino Community (Chapter 2)Differences in Presentation of CTCL (Chapter 3)Personalized...
by CheckRare Staff | Mar 14, 2023
« Prev1 / 1Next »CTCL: Criteria to Diagnose, Counsel Patients, and Work-Up for Early-stage Mycosis FungoidesCriteria to Diagnose Mycosis Fungoides (Chapter 1)Counseling Patients With T-Cell Dyscrasias (Chapter 2)Work Up for Early Stage Mycosis Fungoides...
by CheckRare Staff | Mar 11, 2023
The US Food and Drug Administration (FDA) has approved trofinetide (Daybue) to treat persons with Rett syndrome who are two years of age and older. This the first and only drug approved for the treatment of Rett syndrome. Rett syndrome is a rare progressive...
by CheckRare Staff | Feb 27, 2023
Everett Alms, a 16-year-old with Bardet-Biedl syndrome, along with his parents, Bonnie and Will, describe their diagnostic journey. Bardet-Biedl syndrome is a rare genetic disease that can impede many organs. Numerous genes have been linked to this rare disease...
