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Results For "rare-disease"

212 News Found

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


Novo Nordisk weathers pricing pressure, bets big on oral Wegovy
News | February 05, 2026

Novo Nordisk weathers pricing pressure, bets big on oral Wegovy

Operating profit fell 1% in Danish kroner to DKK 127.7 billion but rose 6% at constant exchange rates


Sanofi’s Venglustat shows breakthrough results in rare neurological Gaucher disease
Clinical Trials | February 04, 2026

Sanofi’s Venglustat shows breakthrough results in rare neurological Gaucher disease

Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs


Budget 2026 -27: Pharma sector cheers Rs. 10,000 crore Biopharma plan
Policy | February 01, 2026

Budget 2026 -27: Pharma sector cheers Rs. 10,000 crore Biopharma plan

The Union Budget makes a clear and timely choice by placing biopharma at the centre of India’s next manufacturing wave


Budget 2026–27: Rs. 1,06,530.42 crore allocated to Ministry of Health and Family Welfare
Policy | February 01, 2026

Budget 2026–27: Rs. 1,06,530.42 crore allocated to Ministry of Health and Family Welfare

The Union Budget 2026–27 accords strong priority to the upgradation and expansion of healthcare infrastructure and medical education


Biocon Biologics gets credit boost from S&P, outlook stable
News | January 30, 2026

Biocon Biologics gets credit boost from S&P, outlook stable

The rating on senior secured notes issued by Biocon Biologics Global PLC was also upgraded to ‘BB+.’


Ionis and Otsuka score EU nod for DAWNZERA, offering new hope for rare HAE patients
News | January 28, 2026

Ionis and Otsuka score EU nod for DAWNZERA, offering new hope for rare HAE patients

The EU approval of DAWNZERA is an important milestone that reflects our ongoing commitment to bring this innovative medicine to people in need across the globe


AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder
Biotech | January 20, 2026

AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder

Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector


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


Bristol Myers Squibb reports positive Phase 3 results for Camzyos in adolescents with oHCM
Clinical Trials | January 14, 2026

Bristol Myers Squibb reports positive Phase 3 results for Camzyos in adolescents with oHCM

The study showed a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo