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Results For "rare-disease"

212 News Found

AstraZeneca expands its portfolio by bringing rare disease therapy for patients in India
News | March 01, 2023

AstraZeneca expands its portfolio by bringing rare disease therapy for patients in India

Receives regulatory approval for its molecule ‘Selumetinib’ in India


Sanofi reaffirms commitment to rare diseases in India
News | February 28, 2023

Sanofi reaffirms commitment to rare diseases in India

Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India


Syntegra partners with TMA Precision to reduce the diagnostic odyssey for rare disease with synthetic data-enabled tools
Digitisation | June 16, 2022

Syntegra partners with TMA Precision to reduce the diagnostic odyssey for rare disease with synthetic data-enabled tools

TMA will leverage Syntegra's synthetic data engine to accelerate the building of predictive diagnostic algorithms


Strengthening the access to rare disease treatment in India
Healthcare | May 09, 2022

Strengthening the access to rare disease treatment in India

Takeda and United Nations Global Compact Network of India (UNGCNI) join hands


NORD announces over US $ 100,000 in grant funding available for rare disease research
Biotech | May 02, 2022

NORD announces over US $ 100,000 in grant funding available for rare disease research

Three RFPs now open for qualified researchers through NORD's Jayne Holtzer rare disease research grants program


Dr Sheela Nampoothiri appointed as co-chair of ICMR's Rare Diseases expert committee
People | March 08, 2022

Dr Sheela Nampoothiri appointed as co-chair of ICMR's Rare Diseases expert committee

Dr Ratna Puri from Sir Ganga Ram Hospital, Delhi is the Chairperson of the expert committee.


Cytel and Pulse Infoframe to transform rare disease research landscape
Biotech | December 10, 2021

Cytel and Pulse Infoframe to transform rare disease research landscape

Both companies will combine their expertise to channel real-world evidence toward ground-breaking new treatments in rare diseases


ENSPRYNG cuts MOGAD relapse risk by 68% in landmark Phase III trial: Roche
Clinical Trials | April 22, 2026

ENSPRYNG cuts MOGAD relapse risk by 68% in landmark Phase III trial: Roche

The Phase III METEOROID study met its primary endpoint, with ENSPRYNG cutting the risk of a first relapse by 68% compared to placebo in adults and adolescents


Ultomiris shows strong interim results in Phase III IgA nephropathy trial
Clinical Trials | April 22, 2026

Ultomiris shows strong interim results in Phase III IgA nephropathy trial

IgAN is a rare, progressive kidney disease driven by abnormal IgA immune complexes that deposit in the kidneys


Aurobindo Pharma bags USFDA nod for Glycerol Phenylbutyrate Oral Liquid
Drug Approval | April 20, 2026

Aurobindo Pharma bags USFDA nod for Glycerol Phenylbutyrate Oral Liquid

The approved product is a generic version of Ravicti, originally developed and marketed by Horizon Therapeutics