Doctors Said These Women’s Mutated Genes Wouldn’t Harm Them
The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...
Hope College21h
Lecture Series to Debut with “Gene Therapy 101 – What you should know”
The new Rodger Kobes and Michael Keller Science and Human Health Lectureship at Hope College will debut with the general-audience address “Gene Therapy 101 — What you should know” by Dr. Anne Connolly ...
biopharma-reporter5d
FDA designations boost AI-driven cell therapy approach for Duchenne muscular dystrophy
The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...
Business Wire3d
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...
kpax1d
Calves to Cure helps fund research for Montana boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...
WTVC5d
6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company
Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...
The American Journal of Managed Care5d
Atrial Arrhythmias Common in DMD, but More Data on Treatment Are Needed
Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and ...
Avidity Biosciences Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...
Week In Review: Nippon Shinyaku Signs $735m Deal For DMD Therapy
Japan’s Nippon Shinyaku will acquire European rights for a Duchenne muscular dystrophy disease therapy developed by Capricor ...
Govt to launch a programme to develop 12 indigenous drugs for 8 rare diseases
New Delhi: The Central government is set to launch a scheme to develop 12 indigenous drugs for eight rare diseases, health ...
Managed Healthcare Executive5h
PSG Launches Solution to Assess Payers’ Gene Therapy Risk
GeneCQ uses integrated medical and pharmacy claims data to provide insight into the exposure to the costs for gene therapies.