Life with GNEM: Voices from around the World Lift the Veil on the Patient Experience – Part 1
GNE Myopathy is a rare genetic disease with a worldwide presence. There are currently 2,000* identified cases on 6 continents. The Neuromuscular Disease Foundation [...]
Breaking News for Rare Disease Patients – NDF among 85 Rare Disease Advocacy Groups Urging Senate to Include RARE Act in New Legislation!
The Neuromuscular Disease Foundation (NDF) is proud to be among the eighty five advocacy organizations representing people living with rare diseases that recently came [...]
Consciously Shifting the Dynamic of Patient Storytelling
The Neuromuscular Disease Foundation and RAM Challenge You to Change the Way You Tell Patients’ Stories When the NDF interviews a patient to publish [...]
Sure, You’ve Heard About GNEM. But Do You Know About the Bulgarian Variant?
GNEM, commonly called GNE myopathy, “is a rare (autosomal recessive) genetic disorder that causes progressive skeletal muscle atrophy and weakness.” Previous names of the condition include [...]
GNE Myopathy – Will Today’s Patients See a Treatment That Will End GNEM in Our Lifetime?
By Dana Corddry I’ve known people to almost faint at the mention of words like “cancer,” “heart disease,” or “dementia,” imagining the uncertainty of one’s [...]
Motherhood Reflections: Exploring Our Journeys & Fostering Emotional Wellness For Ourselves & Our Families With Neuromuscular Disease Foundation’s Emotional Wellness Director Carol Gelbard, LCSW
NDF’s Emotional Wellness Director Carol Gelbard, LCSW, participated in the RARE MAMAS RISING podcast by Nikki McIntosh. The Mother's Day special was released on [...]
What is gene therapy?
Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic [...]
Seeing Clearly: The State of NDF
On Sunday, April 3rd, 2022, NDF held an important webinar, entitled Seeing Clearly: The State of NDF. More than 35 participants from our global [...]
It’s A Marathon, Not A Sprint – Why Is This Ultra Rare Disease So Difficult to Diagnose?
We Talk to Doctor and GNEM Patient Suleyman Kus, about His Journey to Diagnosis, and What the Medical Community Can Do Better.Doctor Suleyman Kus of [...]
How The NDF Maintains Transparency with Patients & Donors
Running a nonprofit is not for the faint of heart. It requires drive, vision, extra doses of passion, resourcefulness, and impeccable communication skills. It [...]