News Medical48m
Characterizing the spectrum of mitochondrial DNA deletion disease syndromes
Researchers from the Mitochondrial Medicine Program at Children's Hospital of Philadelphia (CHOP) have better characterized a spectrum of what were classically considered discrete mitochondrial DNA ...
The Economist1h
CRISPR cures face cost and safety concerns
Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...
Researchers identify novel gene behind neurodevelopmental syndrome for enhanced clinical treatment
A clinical research team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international genetic researchers led a global research study using multi-omics analysis and ...
JD Supra2h
Navigating Key Challenges in M&A Deals in the Rare Disease Sector
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
US health agencies postpone rare disease day
A two-day meeting aimed at raising awareness among policymakers and the public about rare diseases, scheduled for the end of ...
Using genetic technology for rare diseases
It’s a race against a single genetic marker -- and musician turned biotech start-up CEO Casey McPherson says the stakes for ...
My daughter has a rare blood disorder. I combat vaccine skepticism with empathy, not shame | Opinion
Why should you care? The answer is empathy. When we empathize with others, we make decisions that are greater than ourselves.
Frontiers10h
Exploring Innovative Therapies for Rare Inflammatory Skin Diseases
Years of translational research have successfully improved treatment outcomes for common dermatological conditions such as ...
Dairy workers’ cats died from bird flu, but it’s not clear how they got infected
Cats in two separate households died in May after developing severe illnesses that were confirmed as the Type A H5N1 bird ...
BioMarin's Narrow Moat Supported by Rare Disease Therapies and Developing Pipeline
BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...
Advocating for a child with a rare disorder brings heartbreak. And then there’s the joy.
When a child might not be — when something is wrong, yet no one seems to know what it is — the anxiety is soul-crushing. And ...
Premier Construction Magazine1d
Ekho Studio completes 7,000 sq ft office design, bringing Alexion into the AstraZeneca fold at St Pancras Square, London
Ekho Studio completes 7,000 sq ft office design, bringing Alexion into the AstraZeneca fold at St Pancras Square, London ...