Disease Presentation Treatment 
Science Daily: Pharmacology News
JULY 18, 2025
The process, known as pronuclear transfer, was designed to prevent the inheritance of devastating mitochondrial diseases passed down through the mother’s DNA. The early results are highly promising: all the babies are developing normally, and the disease-causing mutations are undetectable or present at levels too low to cause harm.
Drug Target Review
JUNE 9, 2025
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
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FDA Law Blog: Drug Discovery
NOVEMBER 10, 2024
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Chemical Biology and Drug Design
APRIL 4, 2025
At present, there are few studies on the drug resistance of mIDH1 inhibitors. Currently, the FDA has granted approval for the use of the small molecule inhibitor Ivosidenib (AG-120) in the treatment of IDH1-mutated AML and cholangiocarcinoma.
Drug Target Review
JULY 1, 2025
Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They are patient-specific outcome predictions, generated using disease-specific machine-learning models trained on large, longitudinal clinical datasets. Digital twins offer a way forward.
Drug Target Review
APRIL 7, 2025
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.
Drug Target Review
JUNE 16, 2025
In this article, Stephan Schann, Chief Scientific Officer at Domain Therapeutics , explains why this class of drug target is so useful despite the challenges they present. Yet although they are a proven drug target family with widespread utility, they remain largely underexplored for tackling inflammatory diseases.
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