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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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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

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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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.

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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111
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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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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.