Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative ...

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...

Recovery.com, a global platform that connects patients, loved ones, and providers to mental health and addiction treatment options, has released its first-ever State of Recovery report. Based on ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Eko Health, a leader in applying artificial intelligence (AI) for the early detection of heart and lung diseases, today ...

Learn more about whether Ultragenyx Pharmaceutical Inc. or Vericel Corporation is a better investment based on AAII's A+ ...

South San Francisco, CA, January 30, 2025 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded nearly ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

and Arrowhead Pharma’s antisense oligonucleotide ARO-HBV. ARO-HBV was previously partnered with Johnson & Johnson until GSK acquired rights to the drug as a companion for bepirovirsen in a $1 ...

PHILADELPHIA, PA — Imvax, Inc, recently closed a $29 million financing round from existing investors. This marks the latest ...

A team from the University of Geneva and Bayer AG present their findings on the on-off retention of oligonucleotides and the ...

GSK has revealed updated results from its phase 2b trial of its antisense drug for hepatitis ... with 16% of patients on NA therapy and 25% of those taking bepirovirsen achieving clearance of ...