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16 Weird Diseases You Won’t Believe Are Real

Zombies are the stuff of science fiction, right? Well, people who suffer from Cotard’s syndrome are convinced they are dead or, at the very least, their internal organs are failing. They may refuse to ...
STAT

My granddaughter has a rare disease. But clinical trials often exclude patients like her

As the grandfather of a girl with a rare disease, I’ve learned that for families like ours, hope often comes not as a cure, but as a clinical trial: carefully designed but inevitably exclusive. My ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
Hosted on MSN

These 5 Medical Conditions Sound Too Weird To Be True - Pt 1

When it comes to health, most of us are familiar with the usual suspects: fevers, colds, maybe a sprain or two. But the human body and brain are capable of some truly strange malfunctions that sound ...
CNBC

CNBC launches CNBC Cures to help 30 million Americans with rare diseases

CNBC Cures aims to help raise awareness of rare diseases and improve patient outcomes for people living with them. Led by "Squawk Box" anchor Becky Quick, the initiative was inspired by her family's ...
The American Journal of Managed Care

US Spending on High-Revenue Rare Disease Drugs in 2022

One in 5 of the highest-revenue drugs of 2022 was exclusively approved for rare conditions, accounting for more than 7% of US pharmaceutical spending. Study Design: Cross-sectional study. Methods: ...
Medical Xpress

Rare Diseases

A newly developed AI tool can dramatically speed up the search for the genetic causes of rare diseases, a process that often takes years and frequently ends without answers. The tool analyzes how ...
USA Today

Her 5-year-old has a severe, rare disease. There's a cure, but she can't get it.

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...
Los Angeles Times

Contributor: By loosening standards, the FDA isn’t doing rare-disease patients any favors

This is read by an automated voice. Please report any issues or inconsistencies here. If you’re faced with a serious disease, you better hope it’s not a rare one. After an often tortuous path to ...
Business Wire

Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9)

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the ...
Fierce Healthcare

Survey: Many rare disease patients struggle to navigate care

Communication gaps, preventable delays and limited support place significant responsibility on patients on the rare disease journey, a new survey finds. The survey (PDF) was conducted by PANTHERx Rare ...