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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

The therapy is approved to be used in combination with glucocorticoid replacement therapies (steroids) to control androgen levels in adults and pediatric patients four years of age and older with classic CAH. Neurocrines share price increased by six percent after news of the approval.

Genetics 116
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STAT+: Pharmalittle: FDA approves a gene therapy for a rare blood disorder; pharmacy chains ordered to pay $650 million over opioid crisis

STAT News

Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio , STAT tells us. As always, do keep in touch. We relish your insights and tips.

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Top 20 Best-Selling Neurology Drugs to Watch in 2024

XTalks

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. mL, depending on the pharmacy. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. mL) is around $620 for a supply of 0.25

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Covid-19 vaccine success bolsters nanoparticle drug delivery research

Pharmaceutical Technology

Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

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Miplyffa Approved as First Treatment for Niemann-Pick Disease Type C (NPC)

XTalks

NPC is a rare genetic disorder that leads to progressive neurological symptoms and organ dysfunction. The FDA issued a complete response letter to Zevra for its application in which it questioned the interpretability of this scale in terms of its validity and reliability.

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Drug development and working with specialised Rare Disease networks

pharmaphorum

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs.

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REGN-COV2 Independent Data Monitoring Committee Recommends Holding Enrollment in Hospitalized Patients with High Oxygen Requirements and Continuing Enrollment in Patients with Low or No Oxygen Requirements

The Pharma Data

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.