Genetic Disease Archives - Xtalks

Wednesday, August 12, 2026 | 11am EDT: Accelerate Cell Culture Media Development with Automated Cell Analysis

Clinical Trial Design in Wolfram Syndrome: Lessons from HELIOS and Rare Disease Research

Inside the National Society of Genetic Counselors: Expanding Access to Genomic Care

What’s Next for Spinal Muscular Atrophy Research? SMA Europe Weighs In

Otarmeni Gene Therapy Wins Landmark Approval for Hearing Loss

How Genomic Data Improves Drug Development Timelines and Success

World Hemophilia Day 2026: New Treatment Era and Improving Diagnosis

Cytokinetics’ Myqorzo Wins FDA Approval for oHCM, Set to Challenge BMS’s Camzyos

January 23, 2026

Dawnzera (Donidalorsen) Enters FDA-Approved HAE Market with RNA-Targeted Therapy

October 15, 2025

Ekterly (Sebetralstat) Becomes First Oral On-Demand Treatment for HAE

October 14, 2025

Forzinity (Elamipretide) Approved by FDA: A First for Barth Syndrome and Mitochondria-Targeted Therapy

October 10, 2025

Top 5 Best-Selling Genetic Disease Drugs to Watch in 2025

September 11, 2025

Psoriasis Awareness Month 2025: Advancing Research, Treatment and Understanding

Sephience (Sepiapterin) for PKU in Children and Adults: New BH4 Precursor Wins FDA Nod

Business & Finance News

Harliku, First Ever FDA‑Approved Treatment for Alkaptonuria (AKU), Launched in US by Cycle Pharmaceuticals

CSL’s Andembry (Garadacimab) Gets FDA Approval as New HAE Prophylactic Option

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

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