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Results For "rare-diseases"

142 News Found

Novartis locks in $23 billion US expansion with seventh new facility
News | May 05, 2026

Novartis locks in $23 billion US expansion with seventh new facility

The latest addition, a new API facility in Morrisville, North Carolina, will produce key components for solid dosage tablets, capsules, and RNA therapeutics


FDA pushes forward on gene-editing therapies with new safety roadmap
Biotech | April 20, 2026

FDA pushes forward on gene-editing therapies with new safety roadmap

Genome editing holds extraordinary promise for treating previously incurable genetic diseases


HRV Pharma, Shodhana join hands to build next-gen API pipeline for global markets
News | April 07, 2026

HRV Pharma, Shodhana join hands to build next-gen API pipeline for global markets

Multi-year CDMO partnership combines AI-led virtual development, precision chemistry and GMP manufacturing to accelerate five high-science APIs across regulated markets


Sanofi appoints Dr Parikshit Chaudhari as Head of Market Access & Pricing Strategy
People | April 06, 2026

Sanofi appoints Dr Parikshit Chaudhari as Head of Market Access & Pricing Strategy

He brings extensive cross-functional expertise built through leadership roles


FDA nod to first treatment for rare genetic brain disorder
Drug Approval | March 12, 2026

FDA nod to first treatment for rare genetic brain disorder

The approval comes after a systematic review of published literature


UK pushes space-made medicines from orbit to patients with new support package
R&D | March 10, 2026

UK pushes space-made medicines from orbit to patients with new support package

The initiative provides clearer regulatory guidance and a defined path from research in space to patient access on Earth


Merck delivers resilient 2025 performance despite global headwinds
News | March 06, 2026

Merck delivers resilient 2025 performance despite global headwinds

Strong performances in its Life Science, Healthcare and Electronics divisions — particularly Process Solutions, Cardiovascular treatments and Semiconductor Materials — powered the company’s results


Merck restructures Human Health division for future growth
News | February 23, 2026

Merck restructures Human Health division for future growth

Merck is currently conducting approximately 80 Phase 3 studies, and the company expects more than 20 new growth drivers over next several years, almost all with blockbuster potential


Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease
Drug Approval | February 07, 2026

Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease

This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease