YourTango13h
The Symptom I Ignored At Cheerleading Practice Turned Out To Be A Rare, Life-Altering Genetic Disease
FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...
Harvey Weinstein Now Has A Rare Disease, Sources Say
Harvey Weinstein, the Hollywood producer whose predatory behavior sparked the Me Too movement and landed him in prison, has ...
Brain on Fire: The Mysterious Disease That Mimics Mental Illness
Research reveals how antibodies affect brain receptors in patients with anti-NMDAR encephalitis, a condition often ...
Looking back: Schenectady boy's fight against rare disease inspires memorial fund
Fifty years ago, a memorial fund for research in Reye’s disease was being established at Albany Medical College.
7-Year-Old with Rare Disease — 'Basically Childhood Dementia' — Needs $172,000 Therapy Every 2 Weeks (Exclusive)
Emma Austin needs $172,000 therapy every two weeks to slow the progression of Batten disease, which has "no known survivors." ...
STAT7d
The biggest challenge facing the FDA’s new Rare Disease Innovation Hub
Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest and a visiting ...
How a rare disease changed a Troy mom’s life
It was very scary.” They found out she had a rare condition called systemic mastocytosis, a condition that affects every ...
Norway’s crown princess placed on sick leave due to rare lung disease
Norway’s crown princess placed on sick leave due to rare lung disease - The princess must take time off to manage her chronic ...
Business Wire2d
Savara Announces the Rare Lung Disease, Autoimmune Pulmonary Alveolar Proteinosis (aPAP), is Highlighted on a New Episode of “The Balancing Act ® ” Airin…
Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, ...
JD Supra2d
Considerations in View of the Lived Experience of Affected Individuals: Compliance in the Rare Disease Space
This article, written in partnership with members of the Rare Disease Compliance Consortium, focuses on the role of Patient Advocacy Organizations (“PAOs”) and patient advocates as critical ...