Rare Disease Film Festival Coming to NYC

This May 18th, Chelsea in New York will host DISORDER, the rare disease film festival. This will be the third DISORDER festival that provides an opportunity for filmmakers to share their rare disease stories with a live audience of patients, advocates, researchers,...

Rare Disease Patients – Their Time Has Come

  Priya Kishnani, MD, Duke University School of Medicine, speaks to the rare disease patient community and says ‘their time has come’. Dr. Kishnani was speaking largely to the patient advocacy groups associated with lysosomal storage diseases, like Pompe disease,...

Rare Disease Families

  Dianne Linnehan, Director of Operations of the Phelan-McDermid Syndrome Foundation is also the parent of a person with Phelan-McDermid syndrome, and as she explains in this interview, her daughter provides her with immeasurable strength and joy, despite the...

Lipodystrophy Explained

  Andra Stratton, president of Lipodystrophy United provides an overview of lipodystrophy. Lipodystrophy is a rare medical condition in which persons have an abnormal distribution of fat in the body. That abnormality can present itself in many ways. People with...

FARA Focused on Funding Research for Friedreich’s Ataxia

  Brigid Brennan of the Friedreich’s Ataxia Research Alliance (FARA) discusses the origins of this patient organization and its mission to focus on research about Friedreich’s ataxia. Some of the research FARA is funding includes a natural history study to better...

Rare Diseases are a Global Concern

  Femida Gwady-Sridhar, PhD, talks her involvement with Rare Disease International (RDI). RDI is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. As Dr. Gwady-Sridhar explains in this...
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