Shares of gene therapy developer uniQure N.V. (QURE) shot up 41% on Thursday afternoon after it announced that the U.S. Food ...

Qure's AMT-130 receives FDA Breakthrough Therapy designation for Huntington's disease, adding to Fast Track, RMAT, and Orphan ...

State legislature needs to pass legislation to increase funding, and oil and gas companies need to participate in remediation ...

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announces the publication of ...

Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...

Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investorsProceeds to ...

Methods: This study identified the HD-ZIP family using HMM and TBtools, constructed a phylogenetic tree with OrthoFinder, and analyzed gene family expansion and contraction using CAFE. Conserved ...

Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash. Along with ...

Genetics plays a major role in the size of a penis. This includes genes on the X and Y chromosomes that direct the formation of the testicles and penis. Other factors influencing penis size include ...

Eye color genetics is complex, with multiple genes from each parent determining a person's eye color. Scientists once believed a single gene controlled eye color, but research shows that more than 50 ...