Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

Marfan syndrome is a genetic disorder that affects…

UPenn’s Orphan Disease Center Takes a Rare Approach
    Rare disease centers at academic institutions are usually places that focus on early-stage research of faculty members, but the Orphan… Continue Reading
Global Genes Rare Disease Stock Index Falls 7.51% After Tumultuous Week
The Global Genes Rare Disease Stock Index fell 7.51 percent for the week ending February 9, as the market engaaged in wild swings that had… Continue Reading
MHRA Halts Dosing on Sarepta DMD Trial Over Safety Concerns
Rare Daily Staff Sarepta said the Medicine and Healthcare products Regulatory Agency halted dosing in its late-stage U.K. study of… Continue Reading
Video: Love Letters Series I Brick in the Wall
Brick in the Wall follows the life and legacy of Joshua Frase, and his family’s journey of finding a cure for his rare disorder,… Continue Reading
Abeona Says Early Data from Ongoing Study Gene Therapy for Sanfilippo Shows Cognitive Improvement
Rare Daily Staff Abeona Therapeutics said updated clinical data from the ongoing phase 1/2 trial for its experimental gene therapy for the… Continue Reading
The Education of a Young Doctor
After completing a master’s degree in neuroscience, Jessie Kulaga-Yoskovitz needed to find a job before she began medical school. A friend… Continue Reading
Rare Leader: Michael L. Weamer, President and CEO of The Marfan Foundation
The Basics Name: Michael L. Weamer Title: President & CEO Organization: The Marfan Foundation Disease focus:  Marfan syndrome and related… Continue Reading