#rare-diseases

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#clinical-trials #crispr #gene-therapy #patient-advocacy #medical-research #genetic-disorders
Science
fromTechCrunch
1 week ago

How AI is helping solve the labor issue in treating rare diseases | TechCrunch

AI multiplies scientific productivity, automating drug discovery tasks to tackle workforce shortages and accelerate development of treatments for thousands of neglected and rare diseases.
Medicine
from24/7 Wall St.
3 weeks ago

3 Biotech Stocks That Could Double In 2026

Small- and mid-cap biotech stocks with strong clinical catalysts, like Denali, offer potential for large upside in 2026 amid renewed investor interest.
#crispr
fromWIRED
1 month ago
Medicine

Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments

fromWIRED
1 month ago
Medicine

Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments

more#crispr
Medicine
fromwww.theguardian.com
1 month ago

The Guardian view on gene editing: breakthroughs need a new social contract | Editorial

Few genes cause thousands of rare disorders, yet high drug-development costs and weak commercial incentives leave most genetic conditions without scalable therapies.
Public health
fromFast Company
2 months ago

Rare disease: Our $1 trillion healthcare reform opportunity

Implement universal newborn genome sequencing to diagnose rare diseases early, enable effective treatments, reduce long-term healthcare costs, and prevent avoidable child deaths.
Medicine
fromPsychology Today
2 months ago

The Psychological Toll of Living with a Rare Disease

Accurate diagnosis of rare neurodegenerative disorders like progressive supranuclear palsy reduces uncertainty, guides care, and connects patients to resources.
Science
fromWIRED
5 months ago

The Next Era of Gene Editing Will Be Disease Agnostic

A single disease-agnostic gene-editing strategy could treat many unrelated rare genetic diseases, addressing the impracticality of developing bespoke therapies for thousands of conditions.
Medicine
fromFast Company
5 months ago

Here's why genomic sequencing should be a standard of care

Genomic sequencing must become standard of care to accelerate diagnosis and treatment access for pediatric rare disease patients, shortening diagnostic odysseys and saving lives.
Public health
fromwww.independent.co.uk
6 months ago

Boy with rare genetic skin disorder can now play football after breakthrough trial

A clinical trial has offered a 12-year-old boy with RDEB the opportunity to engage in sports like football and biking.
fromsiliconvalleyjournals.com
6 months ago

Citizen Health Raises $30 Million in Series A to Launch AI Advocate for Every Patient

With rare diseases affecting over 400 million people worldwide and 95% lacking approved treatment, the urgency of Citizen's mission cannot be overstated.
fromHarvard Gazette
6 months ago

A setback to research that offered hope for Fibrous Dysplasia patients - Harvard Gazette

This funding was more than just a financial award-it was a crucial investment in understanding and eventually treating a devastating disease. Advancing research in FD/MAS benefits those living with this rare disease and holds great potential for broader medical applications.
Health
Fundraising
fromwww.newsshopper.co.uk
7 months ago

'My son's spine twists and crushes his organs - it's a race against time to save him'

Harley, diagnosed with a rare skeletal condition, faces severe spinal deterioration requiring urgent treatment.
#genetic-disorders
SF parents
fromwww.bbc.com
8 months ago

Parents of kids with rare gene disorder raise 70k

Mel and Charlie are raising funds to support research for a rare genetic disorder affecting their children through organized charity events.
US news
fromBoston.com
9 months ago

Baby is healed with world's first personalized gene-editing treatment

A custom gene-editing treatment for a rare genetic disorder may pave the way for therapies for thousands of other genetic diseases.
SF parents
fromenglish.elpais.com
9 months ago

A Spanish therapy saves the lives of the unluckiest family in the world'

A revolutionary gene therapy has transformed the lives of children suffering from a rare genetic disorder caused by a DNA mutation.
more#genetic-disorders
Parenting
fromwww.bbc.com
8 months ago

'I'm going to outlive our child, there are no words'

Parents of a child with a rare, unnamed genetic condition face profound emotional challenges and seek connection with others in similar situations.
fromSilicon Canals
9 months ago

Dutch-based Azafaros raises 132M

Azafaros has been impressive in its execution with nizubaglustat poised to begin Phase 3 clinical development and the potential to significantly improve the lives of NPC and GM1/GM2 patients.
Startup companies
OMG science
fromwww.independent.co.uk
9 months ago

Rare diseases often go undiagnosed or untreated in parts of Africa. A project seeks to change that

The article illustrates the impact of rare diseases on families, highlighting the emotional toll and challenges faced in accessing timely medical care.
OMG science
fromNature
10 months ago

Whole-genome sequencing susses out rare diseases

Whole-genome sequencing offers a more comprehensive solution for diagnosing rare genetic diseases than traditional exome sequencing.
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