Opus Genetics Gene Therapy Accepted into FDA's Rare Disease Program

The Food and Drug Administration accepted Opus Genetics's investigational gene therapy into its Rare Disease Evidence Principles program, the company said Monday. The clinical-stage biopharmaceutical ...

Hantavirus: What to know about rare disease on Atlantic cruise ship

Three people aboard a cruise ship sailing the Atlantic Ocean have died as a result of suspected hantavirus infections, according to the World Health Organization. Hantaviruses are a family of ...
News-Medical.Net

Advanced AI models outperform pediatricians in diagnosing rare diseases

Artificial intelligence (AI) is increasingly being explored as a tool to support clinical decision-making, yet its real-world performance in pediatric diagnosis remains unclear.
BioSpace

Chiesi snaps up KalVista for $1.9B, betting big on rare disease med Ekterly

Chiesi Group is taking KalVista Pharmaceuticals under its wing, paying $1.9 billion for the biotech’s oral therapy Ekterly to ...
BioSpace

AstraZeneca prunes asthma, rare disease pipeline, cultivates cancer portfolio

While AstraZeneca has discontinued work on four assets—including one in asthma and another in acromegaly—the pharma has also ...
Fierce Biotech

Astellas thins early pipeline, swaps out struggling rare disease gene therapy

Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...
Hosted on MSN

AI is changing rare disease diagnosis forever

Rare disease patients often face years of uncertainty before getting a diagnosis, but new AI systems like DeepRare are cutting that wait. By combining symptoms, genetic data, and medical literature ...
Fierce Pharma

Chiesi lays out $1.9B to bolster rare disease offerings with KalVista buyout

As Ekterly nears the one-year anniversary of its FDA approval, Italy’s Chiesi Group is making moves to acquire the rare ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
Hosted on MSN

How AI is reshaping rare disease diagnosis

A groundbreaking AI system, DeepRare, has outperformed rare disease specialists in accurately identifying complex conditions, offering hope for faster diagnoses. Rare diseases often take years to ...
STAT

My granddaughter has a rare disease. But clinical trials often exclude patients like her

As the grandfather of a girl with a rare disease, I’ve learned that for families like ours, hope often comes not as a cure, but as a clinical trial: carefully designed but inevitably exclusive. My ...
CNBC

A lost voice, a rare disease and the family conversations that followed

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases. Here are some of the stories we received. Living with a rare disease ...