Rare Disease Report®

FDA Approves Viltolarsen for Duchenne Muscular Dystrophy

August 12, 2020

Article

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

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.

x