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FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary. The gene presents a complex landscape. million DNA bases.

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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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Researchers reprogram gene therapy viral vectors to bind specific protein targets

Broad Institute

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

A condition of approval is the completion of a Phase 3 study designed to evaluate pheno-conversion in SOD1 mutation carriers (ATLAS study) to confirm clinical benefit. Moreover, exploring the repurposing of existing drugs offers additional pathways to potentially effective treatments through adopting a horizontal thinking-based approach.

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

While treating rare diseases presents significant challenges due to small patient populations and limited financial incentives, the Orphan Products Grants Program provides crucial financial support to help bridge the gap between early research and successful treatment development. Relative to other areas of medicine (e.g.,

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A Potential Antidote for both Azide and Cyanide Poisonings [Toxicology]

ASPET

There do not appear to be any established therapeutics for treating azide poisoning at this time and presently available antidotes to cyanide poisoning are far from ideal, being particularly impractical for use if multiple victims present. Groups of animals challenged with an essentially lethal dose of NaCN (1.5

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FDA Approves J&J’s Darzalex Faspro for Rare Light Chain Amyloidosis

The Pharma Data

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock. It was developed with Genmab.