Written by Rohan Narayanan on August 16, 2021
Research study is open to participants worldwide to advance understanding and treatments for Metachromatic Leukodystrophy, a recessive genetic disorder that is characterized by the accumulation of fats (lipids) called sulfatides causing damage to the nervous system
Washington, DC, August 16, 2021—Cure MLD, an initiative of The Calliope Joy Foundation, and the… Read More
Written by Valaree DonFrancesco on November 10, 2020
Washington, DC, November 10, 2020—This year, in conjunction with its rare disease community partners, the National Organization for Rare Disorders (NORD®) continued to drive research and innovation through the IAMRARE™ registry program. Having launched its first study in 2014, today IAMRARERead More
Written by Valaree DonFrancesco on October 1, 2020
Washington, DC, 10/1/2020 — Today, the National Organization for Rare Disorders (NORD®) opened registration for a natural history study on metachromatic leukodystrophy (MLD), a lethal rare disease currently without an FDA–approved treatment. NORD’s NaturalRead More
Written by Valaree DonFrancesco on November 12, 2019
I attended the NORD 2019 Summit in Washington, DC a few weeks ago. It was my first chance to attend this conference organized by the National Organization for Rare Disorders and, admittedly, the first time I considered going. I had the impression that NORD was a policy and advocacy… Read More
Written by Lisa Sencen on May 20, 2019
Houston native Deborah Skolaski is the loving grandmother of Cori, a seven year-old living with metachromatic leukodystrophy. MLD is a genetic disorder that affects the nerves, muscles and other organs, as well as behavior. Deborah felt helpless as she observed the struggles that her son, daughter-in-law and Cori… Read More
