US approves first gene therapy for children with rare genetic disease
Orchard, which was acquired by Japanese pharmaceutical firm Kyowa Kirin for $477.6 million last year, said it will provide details…
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Browsing Tag
rare diseases
Innovations paving the way for drug discovery of rare diseases
Sanjay Vyas, Executive VP and MD, Parexel examines the challenges associated with rare diseases in India, and emphasises on the…
India’s fight against rare diseases needs more focus on awareness and diagnosis: GlobalData
In November 2023, the Indian Union Health Ministry authorised the sale of four generic medications for the treatment of rare…
Our collaborations with Govt of India focus on building resilient healthcare systems
Sylvia Varela, AVP Asia Area, AstraZeneca discusses about the company’s plans to drive market growth, address public health…
Zydus gets Orphan Drug Designation from USFDA for ZYIL1 to treat Cryopyrin Associated Periodic…
Orphan drug designation by USFDA for ZYIL1 provides eligibility for certain development incentives, including tax credits for…
Xentria gets US FDA orphan drug designation for XTMAB-16 in sarcoidosis
The company is focused on developing novel biologics and biosimilars
AstraZeneca to acquire Alexion
Subject to approvals, the acquisition is expected to close in Q3 2021
US FDA awards six clinical trials research grants to develop medical products for rare diseases
FDA received 47 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than…
Piramal Pharma Solutions to join hands with Epirium Bio to develop and manufacture orphan drugs
The integrated programme, involving three Piramal Pharma Solutions sites, encompasses formulation development, supply of APIs and…
US FDA approves Viltepso injection for treatment of Duchenne muscular dystrophy mutation
The approval was granted to NS Pharma