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. 2 This progress has implications for diagnosis, therapeutic efficacy, and potentially establishing clinically relevant endpoints.
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.
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thought leadership
FEBRUARY 26, 2025
Rare diseases are defined in the Orphan Drug Act as diseases or conditions that affect less than 200,000 people in the United States. Evaluating and developing treatments for rare diseases remains a key priority for FDA and is incentivized through the Orphan Drug Act.
PPD
SEPTEMBER 16, 2024
Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease.
PPD
MARCH 13, 2024
In every early phase clinical trial, the transportation, chain of custody, environmental monitoring and subsequent analysis of biological samples are the cornerstones of a successful, robust and quality outcome. Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy.
Covalent Modifiers
MAY 9, 2024
4c00220 Multiple sclerosis (MS) is a chronic disease with an underlying pathology characterized by inflammation-driven neuronal loss, axonal injury, and demyelination. George Vandeveer, Ti Wang, Zain Yousaf, Christopher J. Helal, and Brian T. Hopkins Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.4c00220
Drug Target Review
OCTOBER 11, 2023
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
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