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Rare Disease Report®
DDP-4 Inhibitors Increase Risk of Bullous Pemphigoid
August 05, 2020
Article
Older, white patients and those who use linagliptin are at a higher risk of bullous pemphigoid.
FDA Approves Epidiolex CBD to Treat Seizures Associated with Tuberous Sclerosis Complex
August 03, 2020
Article
The FDA approved the CBD solution following the results of a randomized, double-blind, placebo-controlled trial.
How Voxelotor Benefits the Blood Shortage
August 02, 2020
Video
The pandemic has burdened blood drives nationally. A new sickle cell drug keeps patients from needing as many transfusions.
Voxelotor in Sickle Cell Disease
July 29, 2020
Video
How has the new agent fared in its indicated patients in its first year on the market?
Gene Therapy Shows Early Potential in XLRP
July 25, 2020
Podcast
New phase 1/2 data shows AAV-RGPR may benefit a previously untreated population of inherited retina disease boys and young men.
The Effects of Sickle Cell Disease Disparity
July 23, 2020
Video
How a decades-long issue has resulted in at-need patients lacking confident, available physicians.
Cannabis and Placebo Similar in Reducing Pain in Sickle Cell Disease Patients
July 17, 2020
Article
The findings suggest cannabis should be further investigated as an alternative to opioids.
FDA Committee Signs Off on Terlipressin for HRS-1
July 16, 2020
Article
The 8-7 decision allows Mallinckrodt to move forward with a Sept. 12 PDUFA date for the investigational agent.
Half of Sickle Cell Patients Dissatisfied with Emergency Department Care
July 13, 2020
Article
A study shows surveyed patients were nearly twice as likely to be satisfied with the care they receive from a usual clinician than an emergency caregiver.
Point-of-Care Newborn Testing Could Improve Pediatric Sickle Cell Care
July 10, 2020
Article
A streamlined testing procedure in sub-Saharan Africa shows improved infant and newborn patient identification.