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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 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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A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza.

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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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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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Antibody-drug conjugates payloads: then, now and next

Drug Target Review

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored.

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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