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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.

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How epilepsy researchers are moving the needle past anti-seizure treatments

Pharmaceutical Technology

This is particularly the case for pediatric forms of epilepsy where gene editing techniques like Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are becoming more important, says Ferraro. GW Pharmaceuticals’ Epidiolex (cannabidiol), the first-FDA approved CBD treatment , is also a valuable addition, he adds.

Research 264
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World AIDS Day 2023: New and Promising Treatments for HIV/AIDS

XTalks

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. At the moment, the only FDA-approved treatments for ATTR cardiomyopathy are Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), which were given a green light by the agency two years ago.

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Beqvez Becomes Pfizer’s First Approved Gene Therapy After Nod from Health Canada

XTalks

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Worldwide Clinical Trials

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. Additional research is underway using gene editing via CRISPR, primarily investigating genes with known relationships to ALS, such as SOD1 and C9orf72.

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Vutrisiran Reduces Heart Risks for ATTR-CM Patients in Alnylam’s HELIOS-B Trial

XTalks

The HELIOS-B results build on earlier success from the HELIOS-A trial, which led to the US Food and Drug Administration (FDA) approval of vutrisiran (Amvuttra) for polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

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