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Results For "rare-diseases"

128 News Found

Metropolis Healthcare launches Genomics Centre to scale precision diagnostics and research
R&D | January 14, 2026

Metropolis Healthcare launches Genomics Centre to scale precision diagnostics and research

Backed by two CAP - accredited genomics labs in Delhi NCR and Mumbai, supporting oncology, reproductive health, neurology, transplant immunology, and rare diseases


Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan
News | December 30, 2025

Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan

The partnership also sets the stage for broader collaboration on rare disease treatments


EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP
Drug Approval | December 27, 2025

EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation


Pharma overhaul: EMA hails “Once-in-a-Generation” reform
Policy | December 16, 2025

Pharma overhaul: EMA hails “Once-in-a-Generation” reform

The reform is set to modernise how medicines are developed, authorised, and made available across the EU


Sanofi secures two breakthrough nods in China for rare blood disorders
News | December 16, 2025

Sanofi secures two breakthrough nods in China for rare blood disorders

Qfitlia is the first antithrombin (AT)-lowering therapy approved for routine prophylaxis in hemophilia


Debiopharm launches pivotal phase III trial for novel quarterly acromegaly treatment
Clinical Trials | December 07, 2025

Debiopharm launches pivotal phase III trial for novel quarterly acromegaly treatment

Acromegaly, a rare chronic endocrine disorder caused by excess growth hormone, is seeing promising advances in treatment development


FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma
Drug Approval | November 12, 2025

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition