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

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 ...

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

DelveInsight’s Advanced Cervical Cancer pipeline report depicts a robust space with 70+ active players working to develop 75+ ...

"Imvax secures $29m to support Phase IIb glioblastoma therapy trial" was originally created and published by Clinical Trials ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

The Massachusetts based biotech will now press ahead with human trials for its antisense oligonucleotide (ASO) therapy, ...

The methods and protocols generated in this study are accessible and can be implemented in any standard research laboratory without the need for ...

The FDA is putting Atara’s active Investigational New Drug applications on hold due to manufacturing concerns at a ...

Analysts view setrusumab as a potential $1 billion drug, which could be transformative for Ultragenyx's financial outlook. GTX-102, an antisense oligonucleotide therapy for Angelman syndrome, is ...

This is the latest venture for Amylyx into the ALS field after its previous therapy Relyvrio was pulled from the market in 2024.

Massachusett-based Stoke Therapeutics STOK is making rapid progress with the development of zorevunersen (STK-001), its investigational candidate for treating Dravet syndrome, a severe and progressive ...