The FDA’s new leadership must give rare disease patients a clearer path

At her confirmation hearing, Dr. Heidi Overton, President Donald Trump’s nominee to lead the Food and Drug Administration, ...

Parents of 4-Year-Old with Ultra-Rare Genetic Disorder Are Determined to Give Him as ‘Normal a Childhood as Possible’ (Exclusive)

“I just want people to see Luca as a little boy first,” Mariah George tells PEOPLE. “His diagnosis is a part of his story, ...

Boy, 13, Diagnosed with Rare Genetic Disease Has to Consume Raw Cornstarch to Survive (Exclusive)

While Jen McGowan describes her son as a “really happy baby,” she admits to PEOPLE that his younger years were “intense” ...

Social Media Star Elis Lima Carneiro, 5, Dies from Rare Aging Syndrome

Elis Lima Carneiro, a social media star who shared her life with a rare aging syndrome, died on Sept. 30. She was 5.
BIOENGINEER

Rare Fungal Brain Infection Reveals Striking Christmas-Tree Sign on MRI

A rare and devastating fungal infection of the central nervous system has been documented in striking detail by radiologists ...
ainvest

The Rare-Disease Grant You Can't Buy Is Pointing at One You Can

The appendix grant is a specimen of exactly that mechanism, not the catalyst itself. The dollar amount is immaterial; what is ...
UNILAD

Social media star Elis Lima Carneiro has died aged 5 from rare aging syndrome

Brazilian social media star Elis Lima Carneiro, whose family documented her life with a rare aging syndrome has tragically ...

A story about my childhood, growing up without knowing I had a rare disease (A20 haploinsufficiency)

Well, regarding the symptoms and daily life of my childhood that I wrote about briefly before.First of all, my allergies were ...

I've never felt so helpless: Pennsylvania mother shares baby's battle with rare disease

A central Pennsylvania baby is battling a rare genetic disease, and now her mother is sharing their journey in hopes of ...
BIOENGINEER

Rare Castleman Disease Masquerading as Tuberculosis Finally Diagnosed as TAFRO Syndrome

A 21-year-old man who spent three years battling unexplained fevers, swollen lymph nodes, and dramatic weight loss has become the subject of a remarkable case report that sheds light on one of ...
Zamin.uz

Five-year-old blogger who battled rare disease passes away

Five-year-old Brazilian social media star Alice Lima Carneiro has passed away after battling a rare aging syndrome. Along ...
Daily Mail

How your annual flu jab may help fight cancer, heart disease and other chronic illnesses: As demand for shingles vaccine soars amid evidence it could ward off dem…

It has been billed as one of the biggest medical discoveries in recent history. The shingles vaccine, designed to protect ...