This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

Collaborations Pharmaceuticals, Inc. demonstrates a treatment for CLN1 Batten disease crosses the blood-brain-barrier

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...

US FDA places clinical hold on Regenxbio's rare disease program

Jan 28 (Reuters) - Regenxbio said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its ...
New Atlas

Surprising way cancer cells clear problematic proteins in the brain

For some time, we've known that it's rare to see people with both cancer and Alzheimer's disease. Now, scientists believe ...

In rare cases, autoantibodies can cause severe reactions to a discontinued Chikungunya vaccine

A new study published in the Proceedings of the National Academy of Sciences suggests that rare cases of brain inflammation ...
New Scientist

Why people can have Alzheimer's-related brain damage but no symptoms

Some people don’t develop dementia despite showing signs of Alzheimer’s disease in their brain, and we're starting to ...
News-Medical.Net

New discovery may aid in early detection of rare genetic disease

Virginia Tech researchers discovered an indication hidden in the brain that may help doctors identify children suffering from ...
STAT

FDA halts gene therapy trials after brain tumor found in patient

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

A skin biopsy can detect a rare neurodegenerative disease

By determining the structure of the deposits responsible for transthyretin amyloidosis through a simple skin biopsy, scientists at UNIGE are paving the way for a new diagnostic method for ...
PharmExec

Bringing Huntington’s Story to the Public: Q&A with Heather DeMyers

Teva's Honestly HD campaign, featuring Will Forte, aims to raise awareness about Huntington's disease through personal ...
BlackDoctor.org

Different Types of Brain Tumors: What They Are, How They Present and What To Expect

Brain tumors are abnormal growths of cells in or around the brain. They can be primary (originating in the brain) or secondary (metastatic, spreading to the brain from cancer elsewhere). They can also ...

A rare condition left a Houston student with seizures and psychosis. Why was her 'brain on fire?'

A Texas college student spent months suffering from unexplained seizures and psychosis before UTHealth Houston doctors determined she had a rare condition.