News & Articles from Neuromuscular Disease Foundation
Gene Therapy Effort for GNE Myopathy/HIBM
What is GNE Myopathy? GNE Myopathy (GNEM), also known as HIBM, is a rare genetic muscle disease that occurs in Iranian Jewish communities, as [...]
May 2025 6SL Update
May 2025 6SL Update Dear GNEM/HIBM community, I hope this message finds you well. The NDF executive team has been diligently researching options for [...]
Happy Holidays! Let’s Donate to NDF for Giving Tuesday.
We’re Bringing Unprecedented Scientific Advancements & Remarkable Progress toward Globally Accessible GNEM Therapies into 2025. At the Forefront of the Pre-IND race, We’re Inviting [...]
Neuromuscular Disease Foundation Scientific Advisory Committee Update on 6SL
The Neuromuscular Disease Foundation Scientific Advisory Committee examined the peer reviewed research paper entitled “Pharmacokinetics and clinical efficacy of 6’-sialyllactose (6 SL) in patients [...]
29 February is Rare Disease Day
GNE Myopathy affects people around the globe. NDF continues to support patients and strive for an effective treatment for GNEM. During February, we want [...]
NDF 2023 Grant Updates
We work year-round to enhance the quality of life for those living with GNE Myopathy by funding critical research focused on treatments and a [...]
The Neuromuscular Disease Foundation Leads in Patient Resources and Grant Funding in Service of Rare Disease GNEM Patients Worldwide
Since our inception, NDF has delivered more than ten million dollars in grant funding to the world’s leading rare disease researchers, drug development pharmaceutical [...]
New Patient Care Partner Support Group Programs
The Neuromuscular Disease Foundation (NDF) is proud to offer a broad range of services, support programs and free resources to its global community of [...]
Is Your Confidence Affected by Your Disability?
Confidence is a make-it-or-break-it quality in life. It allows us to stretch our horizons, try new things, meet new people and visit new places. [...]
How Two Young Disability Inclusion Advocates Are Building a More Inclusive World, One Lesson at a Time
By Dana Corddry If you haven’t heard of Eva and Amaira Deotale before, it was only a matter of time. These two mini powerhouses [...]
