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Results For "rare-disease"

198 News Found

USFDA approves first treatment option for generalized pustular psoriasis flares in adults
Drug Approval | September 03, 2022

USFDA approves first treatment option for generalized pustular psoriasis flares in adults

Spesolimab is a monoclonal antibody that inhibits interleukin-36 (IL-36) signaling and is the first treatment specifically approved for the treatment of generalized pustular psoriasis flares


Funds for R&D in drug discovery and manufacturing :PIB
Policy | July 26, 2022

Funds for R&D in drug discovery and manufacturing :PIB

An outlay of Rs. 1,500 crore has been approved for strengthening/ up-gradation of these NIPERs for the period 2021-22 to 2025-26.


H Clinical adds commercial home health capabilities for pharma industry
News | July 01, 2022

H Clinical adds commercial home health capabilities for pharma industry

H Clinical is the leader in decentralized clinical trial support and home visits in Latin America


DTx Pharma appoints Peter Condon as Chief Business Officer
People | June 29, 2022

DTx Pharma appoints Peter Condon as Chief Business Officer

Condon joins DTx Pharma with more than 20 years of experience in business development, commercial planning, and operations.


Nuvectis announces positive data for NXP900 in a preclinical model of Group 4 medulloblastoma
News | June 14, 2022

Nuvectis announces positive data for NXP900 in a preclinical model of Group 4 medulloblastoma

Group 4 Medulloblastoma is a rare disease that mostly affects pediatric patients, in which aberrant SRC signaling was identified as a hallmark of the disease


Medison Pharma announces multi-regional agreement with argenx to commercialize Efgartigimod
News | June 08, 2022

Medison Pharma announces multi-regional agreement with argenx to commercialize Efgartigimod

Efgartigimod is approved in the United States as VYVGART for the treatment of adults with generalized myasthenia gravis


Merck KGaA to expand its membrane and filtration manufacturing facility in Ireland
News | May 23, 2022

Merck KGaA to expand its membrane and filtration manufacturing facility in Ireland

The company will invest approximately €440 million to increase membrane manufacturing capacity in Carrigtwohill and to build a new manufacturing facility at Blarney Business Park, both in Cork, Ireland


Soligenix receives US patent allowance for its thermostabilized vaccine platform
Biotech | May 23, 2022

Soligenix receives US patent allowance for its thermostabilized vaccine platform

Includes thermostabilized Ebola vaccines


Don’t be left short-changed post approval – plan in change orders early for accelerated pathways: Stephanie Gaulding
News | May 19, 2022

Don’t be left short-changed post approval – plan in change orders early for accelerated pathways: Stephanie Gaulding

A post approval change order is often necessary, but innovators should plan and prepare for this pre-approval. Planning alongside outsourcing partners will help ensure that only a small-scale study is needed—not a new full clinical study