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, ...
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 ...
This internationally recognized NJ doctor is on the frontlines battling rare diseases
A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...
Her Baby Had to Be Revived 8 Times in the NICU. Now She's Talking About His Rare Condition to Help Moms Like Her (Exclusive)
Audrey Kandil tells PEOPLE her son, Rami, was born with cerebrocostomandibular syndrome, which is a rare genetic disorder ...
Her son has a rare genetic disorder, so this Connecticut mom is taking fight for funding to D.C.
Families in Connecticut and beyond are looking for answers when it comes to treating Angelman Syndrome, but fear funding ...
STAT5d
Soleno Therapeutics’ ‘life-changing’ rare-disease drug approved
The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...
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 ...
Ontario Brain Institute partners with EpiSign to advance diagnosis of rare genetic disorders
The Ontario Brain Institute (OBI) is pleased to announce a joint new initiative with EpiSign Inc., paving the way for improved diagnosis and care for people with rare neurological genetic disorders.
USA Today21d
What is PolG? The rare genetic disease that ended a young prince's life
Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life. Prince Frederik ...
BioPharma Dive5d
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a ...
Scientists want to fight malaria by poisoning mosquitoes with human blood
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