US FDA approves first treatment for rare genetic disorder Prader-Willi syndrome
The U.S. Food and Drug Administration on Wednesday approved Soleno Therapeutics' drug to treat a rare genetic disorder, ...
US FDA greenlights first-ever treatment for rare genetic disorder
The U.S. Food and Drug Administration approved Soleno Therapeutics' drug to treat a rare genetic disorder on Wednesday, ...
Independent.ie on MSN12 小时
‘Every day counts’ – call to add screening for rare muscle-wasting condition to ...
A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...
Her Baby Had to Be Revived 8 Times in the NICU. Now She's Talking About His Rare Condition ...
Audrey Kandil tells PEOPLE her son, Rami, was born with cerebrocostomandibular syndrome, which is a rare genetic disorder ...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...
AsiaOne1 天
'My skin is nice': Raising awareness for children with rare skin conditions
"Why did my classmate say that my skin is yucky?" asked five-year-old Sophie Goh. It may seem like an innocent question, but ...
Teen and his family learn of his rare genetic disorder, Argonaut syndrome from UW Health
After years of wondering, Treyson Wallace and his family learned he has a rare genetic disorder after advanced genomic ...
Medindia2 天
Rare Disease Drug Turns Human Blood Into Mosquito Poison, Battling Malaria
Nitisinone, a drug for genetic disorders, could help control malaria by making human blood toxic to mosquitoes. Anopheles ...
technologynetworks12 天
Ask Me Anything: Precision Medicine for Rare Genetic Disorders
Rare genetic disorders, often complex and challenging to diagnose, impact millions worldwide. Imagine a future where cutting-edge technology accelerates diagnosis, connects patients to life-changing ...
Mother with rare ALS touts 'miracle drug' that has stopped her disease
Raziel Green, 52, an active runner and mother of two, was diagnosed with a rare form of ALS in 2017. Since starting a ...
Express Healthcare13 天
Rare disease treatment: Look at emerging therapies and medications
Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...