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Results For "rare-diseases"

105 News Found

Lupin signs distribution agreement with Celnova for orphan drug NaMuscla
Supply Chain | September 28, 2024

Lupin signs distribution agreement with Celnova for orphan drug NaMuscla

Agreement enables access of EU-approved treatment for myotonia symptoms in non-dystrophic myotonic disorders to patients from Argentina and Colombia


Biogen completes acquisition of HI-Bio
News | July 06, 2024

Biogen completes acquisition of HI-Bio

Felzartamab demonstrated positive interim results from the Phase 2 IgA nephropathy (IgAN) study


AstraZeneca completes equity investment agreement with Cellectis
News | May 08, 2024

AstraZeneca completes equity investment agreement with Cellectis

Cellectis is also eligible to receive an investigational new drug (IND) option fee and development


Sentynl Therapeutics receives marketing authorization for Nulibry from UK MHRA
Drug Approval | April 22, 2024

Sentynl Therapeutics receives marketing authorization for Nulibry from UK MHRA

NULIBRY is a first-in-class synthetic cPMP substrate replacement therapy to reduce the risk of mortality in patients with MoCD Type A


USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria
News | April 17, 2024

USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria

NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway while maintaining the integrity of the classical pathway required for fending off infections


Sanofi and Cipla enters into distribution partnership to expand reach of CNS portfolio in India
Supply Chain | March 27, 2024

Sanofi and Cipla enters into distribution partnership to expand reach of CNS portfolio in India

Cipla will be responsible for the distribution of Sanofi India's six CNS brands including Frisium


Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder
Drug Approval | March 27, 2024

Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder

First and only long-acting C5 complement inhibitor offers patients with AQP4 Ab+


FDA approves Merck’s WINREVAIR for treatment of pulmonary arterial hypertension
Drug Approval | March 27, 2024

FDA approves Merck’s WINREVAIR for treatment of pulmonary arterial hypertension

WINREVAIR is a breakthrough biologic for this rare, progressive disease