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New avenues for rare disease treatment

Drug Target Review

Most of these conditions are genetic in origin and the majority have no effective treatment. That includes countless rare peripheral diseases, including many for which there are currently no treatments. Autophagy boosters, in contrast, are conventional small molecule drugs.

Disease 113
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Metabolism of 2022 FDA approved small molecule drugs PART 2

Metabolite Tales Blog

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. Dermavant’s tapinarof is one such friend. 8 This is not the only point of interest.

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Metabolism of 2023 FDA Approved Small Molecules – PART 1

Metabolite Tales Blog

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals.

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The power of combinations in blood cancers

Drug Target Review

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. Evolution of Cancer Pharmacological Treatments at the Turn of the Third Millennium. Published 2018 Nov 13.

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Condensate biology: advancing drug discovery for complex diseases

Drug Target Review

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint Therapeutics, a platform drug discovery company founded in 2018, has been at the forefront of this exciting field.

Disease 98
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The evolution of cell therapy to address unmet medical needs

Drug Target Review

Our goal is to stand on the shoulders of giants and advance this exciting and new field of medicine, so that it can deliver valuable treatments to patients. Transplanting cells could prove to be more beneficial than treating a particular condition with a small molecule or an antibody, and that really speaks to the power of cell therapy.

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VALILTRAMIPROSATE

New Drug Approvals

ALZ-801 is a potent and orally available small-molecule β-amyloid (Aβ) anti-oligomer and aggregation inhibitor, valine-conjugated proagent of Tramiprosate with substantially improved PK properties and gastrointestinal tolerability compared with the parent compound. 2018; 32(9): 849–861. [2]. 2018 Mar;57(3):315-333.