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

PLOS: DNA Science

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The gene presents a complex landscape.

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FDA approves Nexviazyme® (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease

The Pharma Data

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. LOPD symptoms may present at any age.

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Addressing the Unique Aspects of Rare Disease Product Commercialization

Fierce BioTech

Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. png Listing Introduction Herspiegel experts share best practices for addressing the unique aspects of successful rare disease commercialization.

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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.

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Regeneron Announces Investor Conference Presentations

The Pharma Data

Cowen Virtual Presentation Series – 2020 IO Next Summit at 1:15 p.m. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. EST on Monday, November 9, 2020. EST on Friday, November 13, 2020. EST ( 12:20 p.m.

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Evaluating the Abuse Potential of Lenabasum, a Selective CB2 Cannabinoid Receptor Agonist [Behavioral Pharmacology]

ASPET

Background: Endocannabinoids, which are present throughout the central nervous system (CNS), can activate CB1 and CB2 receptors. CB1 and CB2 agonists exhibit broad anti-inflammatory properties, suggesting their potential to treat inflammatory diseases. However, careful evaluation of abuse potential is necessary.

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Pfizer’s PAXLOVID™ receives FDA approval for adult patients at high risk of progression to severe COVID-19

The Pharma Data

Pfizer’s PAXLOVIDâ„¢ receives FDA approval for adult patients at high risk of progression to severe COVID-19 Pfizer Inc. 1) “Great advancements have been made in the fight against COVID-19, yet the virus remains a present and unpredictable concern. NYSE: PFE) announced today that the U.S. More than 11.6