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Results For "rare-disease"

234 News Found

Mirum Pharmaceuticals bags Canadian nod for LIVMARLI to treat rare liver disorder
News | February 09, 2026

Mirum Pharmaceuticals bags Canadian nod for LIVMARLI to treat rare liver disorder

LIVMARLI’s new tablet form offers patients weighing 22 kg or more a convenient, one-tablet-per-dose option, complementing the existing 9.5 mg/ml oral solution


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