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Results For "Orphan-Drug"

84 News Found

Novartis acquires DTx Pharma to develop siRNA therapies
News | July 19, 2023

Novartis acquires DTx Pharma to develop siRNA therapies

Deal includes DTx-1252, a potential therapy for the neuromuscular disorder Charcot-Marie-Tooth disease type 1A (CMT1A), and two additional preclinical programs for other neuroscience indications


Wacker Biotech partners with Caeregen Therapeutics on regenerative treatment for vision loss
News | June 28, 2023

Wacker Biotech partners with Caeregen Therapeutics on regenerative treatment for vision loss

As Caeregen’s CDMO partner for CTR-107, Wacker Biotech will produce drug substance at its site in Halle, Germany, and complete clinical trial drug product production at its site in Amsterdam, the Netherlands


Zydus announces Phase IV EVIDENCES-XI trial to generate Real World Evidence of Saroglitazar Mg in NAFLD patients with comorbidities
Clinical Trials | June 10, 2023

Zydus announces Phase IV EVIDENCES-XI trial to generate Real World Evidence of Saroglitazar Mg in NAFLD patients with comorbidities

The primary endpoint is to measure the change in liver stiffness measurement performed by transient elastography from Baseline to Week 52


Galmed Pharmaceuticals forms partnership with OnKai
News | May 08, 2023

Galmed Pharmaceuticals forms partnership with OnKai

Both the companies intend to facilitate faster clinical trial enrollment and provide patients with access to novel medications


Microbion gets US patent for the use of inhaled pravibismane for pulmonary infections
News | April 06, 2023

Microbion gets US patent for the use of inhaled pravibismane for pulmonary infections

Extends patent protection for Microbion's proprietary inhaled pravibismane for the treatment of pulmonary infections to 2039


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


Mandaviya focuses on high value pharmaceuticals and high-end medical devices to reduce the import dependency
Policy | February 22, 2023

Mandaviya focuses on high value pharmaceuticals and high-end medical devices to reduce the import dependency

First release of Rs.166 crores incentives under PLI scheme for pharmaceuticals


Sun Pharma launches SEZABY in the US for treatment of neonatal seizures
News | January 27, 2023

Sun Pharma launches SEZABY in the US for treatment of neonatal seizures

SEZABY is a benzyl alcohol-free and propylene glycol-free formulation of phenobarbital sodium powder for injection


Lupin and Exeltis announce reimbursement approval of NaMuscla in Spain
News | January 10, 2023

Lupin and Exeltis announce reimbursement approval of NaMuscla in Spain

NDM disorders are a group of rare, inherited neuromuscular disorders with an estimated prevalence of 1 case per 100,000 inhabitants in Spain, and are characterized by the inability to relax muscles following voluntary contraction (myotonia)