KOIN Portland on MSN1d
Portugal. The Man highlight rare disease month as bandmember’s daughter battles genetic disease
February is Rare Disease Month, an annual effort to highlight the challenges families can face while navigating rare ...
Hosted on MSN1d
The exceptionally rare disease that causes holes to form in your brain
Creutzfeldt-Jakob disease is an extremely rare and fatal brain-wasting disease that's like a human version of "mad cow." ...
US FDA approves SpringWorks' genetic disorder drug
The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare ...
Gene variant could protect against ultra-rare seizure disorder, fruit fly research finds
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
Child with rare epileptic disorder receives long-awaited diagnosis
Researchers provided a long-awaited and rare genetic diagnosis in a child with Lennox-Gastaut syndrome, a type of ...
Can Your Child Inherit Rare Disorders? Know Why Genetic Intervention Is Important
Your genes play a huge role in what makes and shapes your entire existence. Since you inherit your genes from your parents, ...
Gene Therapy: Revolutionizing Medicine By Tackling Diseases At Their Genetic Roots
Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
Activating Complex Regions of the Genome to Treat Rare Diseases
Researchers find a master epigenetic switch that activates silenced genes to compensate for their missing counterparts in a ...
FiercePharma2d
Amid takeover talk, SpringWorks scores FDA nod for rare genetic disease treatment Gomekli
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
JD Supra2d
FCA Enforcement Risk Remains for Rare Disease Stakeholders
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
Baylor College of Medicine2d
Repair Drive: Precision gene therapy for liver disorders
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Katie Price's son Harvey starts Ozempic injections to manage rare genetic condition
Katie Price has revealed her son Harvey will start taking diet drug Ozempic due to worries over his increasing weight. The ...