Many children in a Brazil town lost the ability to walk, so one scientist set about discovering why.

In one case study, it was found that weekly music therapy sessions helped one ten-year-old child with Batten disease better ...

In the small remote town of Serrinha dos Pintos in Brazil, lots of children were left unable to walk and were losing strength ...

A biologist’s decades-long research in a secluded village led to the discovery of a rare genetic condition and transformed ...

Michelle and Samantha Pearson are a mother-daughter duo diagnosed with NF1-PN, a rare and painful disease that causes benign ...

Marley Mansour, 11, is one of fewer than 100 people in the world with a particular mutation in the NARS1 gene. She's set to ...

Scientists have made an important new discovery in medical research, identifying a rare disease called Lyn kinase-associated ...

Healxan, the Cambridge-based AI-enabled, clinical-stage biotech company dedicated to rare diseases, has appointed Dr ...

Mattie Beacham — who at 2 months old was diagnosed with biliary atresia, a rare disease of the liver and bile ducts — came to ...

While extremely rare, encephalopathy (a condition affecting brain function) triggered by mutations in the UBA5 gene has ...

Back on beat! Through the power of music therapy, Levi Nieto is drumming again. Last summer, an autoimmune disease left him unable to walk or talk.

BlackfinBio Limited has received approval from the FDA to trial a novel therapy for Hereditary Spastic Paraplegia ...