Rare Diseases/Orphan Drugs Archives - Page 3 of 5 - Xtalks

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

Strategies to Educate and Support the Entire Patient Network in Rare Disease, Oncology and Complex Clinical Trials

On Rare Disease Day 2025, We Need More Than You Can Imagine

February 20, 2025

FDA Approves Gomekli as First NF1 Treatment for Adults & Children

February 13, 2025

Clinical Trial News

KalVista Shares New Data on Sebetralstat for Hereditary Angioedema (HAE)

February 13, 2025

Key Strategies for Successful Orphan Drug Launch in Europe

A Rare Look into New Guidances: The Future State of Orphan Drug Development

February 25, 2025

Maximizing Therapeutic Success in Rare Diseases through Basket & Umbrella Trial Designs

February 20, 2025

Pioneering Progress for Rare Lives: Cell and Gene Therapy Trials in Pediatric Rare Disease Populations

February 24, 2025

Novo Nordisk’s Alhemo Gets FDA Nod as First Daily Subcutaneous Injection for Hemophilia with Inhibitors

January 6, 2025

Hansa Biopharma Shares Positive Imlifidase Phase II Study Data in Rare Autoimmune Disorder

December 31, 2024

Clinical Trial News

Govorestat Hits Roadblock: FDA Denies Approval for Applied Therapeutics’ Rare Disease Drug

November 29, 2024

Clinical Trial News

Axsome Therapeutics’ Reboxetine Promises Long-Term Symptom Relief in Narcolepsy

November 28, 2024

Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

November 14, 2024

Innovative Clinical Biomarkers for SLE and RA: Advancing Autoimmune Diagnostics

December 12, 2024

Hympavzi (Marstacimab): FDA Approves First Once-Weekly and Pfizer’s Second Hemophilia Therapy

October 16, 2024

Navigating the Complexities of Rare Disease — A Quest Diagnostics Perspective

September 6, 2024

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