Browsing: rare

BridgeBio Pharma BBIO is building its investment case around rare diseases where diagnosis, targeted treatment and commercial access are changing quickly

Bansi Nagji discusses the shifting science behind rare and orphan drug development, including what it takes for therapies to succeed well beyond launch

ADA TOWNSHIP, Mich. — Most mornings, many people move from one task to the next without much thought. But for someone living with multiple system atrophy, those everyday moments can become some of life’s biggest challenges

Sony Interactive Entertainment is offering an extremely rare 50% discount on PlayStation Plus subscriptions to select users who are canceling their membership. While this is a targeted offer as always, a 50% discount is almost unprecedented in this console generation

WASHINGTON, July 7, 2026 /PRNewswire/ — Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to VCA-894A, Vanda’s investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disorder. The designation…

Bansi Nagji discusses the shifting science behind rare and orphan drug development, including what it takes for therapies to succeed well beyond launch

The arXiv:2312.15320GestaltMML paper was revised on July 6, 2026 and describes a Transformer-based multimodal model for rare genetic disease diagnosis. It combines frontal facial images, demographics and clinical text or HPO terms, and the authors report evaluations across 528 GestaltMatcher Database diseases plus several syndrome-specific cohorts. For practitioners, the important signal is that multimodal fusion…

Vertex PharmaceuticalsVRTX announced that it has agreed to acquire commercial-stage company Crinetics PharmaceuticalsCRNX. Per the terms, the large-cap biotech will acquire all outstanding shares of CRNX for $85 per share, valuing the deal at around $10 billion

Rare diseases, individually uncommon but collectively significant, affect fewer than five in 10,000 people per condition. However, with over 7,000 identified rare diseases, the combined impact is substantial – touching around 3.5 million people in the UK and more than 300 million worldwide. This scale underlines the critical need for global partnerships to accelerate both…

Expertise regarding these diagnoses is often limited among healthcare professionals. Furthermore, the lack of harmonised diagnostic coding in health information systems can result in delayed diagnosis, fragmented care, and suboptimal treatment.