The Critical Need to Address Mental Health in the Rare Disease Community, Upcoming Webinar Hosted by Xtalks

Patients and caregivers navigating rare diseases often face emotional strain as significant as the physical symptoms ...

New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for ...
Fierce Biotech

Applied Tx lays off 46% of workforce as rare disease drug approval looks out of reach

While the board considers which path to take, it is implementing various “cost-containment and cash conservation measures,” ...
Medical Xpress

Study supports wider use of FDA-approved drug for rare muscle disease

UNC School of Medicine researchers have led an international clinical trial of patients with generalized myasthenia gravis, finding the therapy is safe and effective across all subtypes. Generalized ...
Seeking Alpha

Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping

SAN DIEGO, Feb. 20, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ...
USA Today

Swiss drugmaker Novartis to buy Avidity Biosciences for $12 billion

Swiss drugmaker Novartis on Sunday said it agreed to acquire U.S. biotech firm Avidity Biosciences for about $12 billion in cash, as the company looks to bolster its portfolio of treatments for rare ...
El Mundo on MSN

A 'genetic master key': creating a therapy capable of treating thousands of rare diseases in one go

Scientists from MIT and Harvard present a new strategy called PERT in 'Nature'. Instead of correcting gene by gene, they use ...
Psychology Today

The Psychological Toll of Living with a Rare Disease

Rare diseases reveal that our sense of identity rests on fragile biological infrastructure, much of it operating outside ...
MyCentralJersey

This internationally recognized NJ doctor is on the frontlines battling rare diseases

Rare Disease Day raises awareness for the thousands of rare diseases affecting millions. Advancements in technology, such as gene therapy, offer hope for potential cures for rare diseases in the ...
Seeking Alpha

Avidity Biosciences Honors Rare Disease Day®

SAN DIEGO, Feb. 29, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ...
The Hearty Soul on MSN

Doctors Warn of Rare Post-COVID Syndrome That Can Be Fatal

At first, it just seemed like a post-pandemic hangover: scattered patients drifting into Yorkshire clinics with damaged lungs ...

What is myasthenia gravis, the rare disease tennis great Monica Seles lives with?

Former tennis star Monica Seles recently revealed she is living with the rare disease myasthenia gravis, which affects 12 in ...