Rare Disease Report®

FDA Approves Viltolarsen for Duchenne Muscular Dystrophy

August 12, 2020


The approval is based on findings from a phase 2 clinical trial.

DDP-4 Inhibitors Increase Risk of Bullous Pemphigoid

August 05, 2020


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


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


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


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


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


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


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


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


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.