FDA approves Novartis' gene therapy for rare muscle disorder

U.S. Food and Drug Administration has approved Novartis' gene therapy for a type of rare muscle disorder, the drugmaker said ...

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 ...

High-intensity training improves muscle function in inflammatory muscle disease, study suggests

High-intensity interval training boosts fitness and muscle endurance more effectively than traditional home exercise programs ...
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 ...
News-Medical.Net on MSN

High-intensity interval training improves fitness in people with inflammatory muscle disease

High-intensity interval training boosts fitness and muscle endurance more effectively than traditional home exercise ...

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 ...
MedPage Today on MSN

FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...
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 ...
Acsh.org

“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases collectively impact millions of Americans, creating a healthcare system that’s ...
Mirage News

Intense Training Boosts Muscle Function in Disease

High-intensity interval training boosts fitness and muscle endurance more effectively than traditional home exercise ...

Canada's $1.5-billion Rare Disease Drug Strategy generating 'return on investment' for patients and society but more needs to be done

Dr. Jennifer Adams, whose daughter was diagnosed at 18 months with PH1, calls Oxlumo a "life-changing medication." As a result, her daughter's "quality of life has improved, hospital visits were ...
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 ...