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Results For "rare-diseases"

128 News Found

Lonza announces first GMP batch completion at next-generation mammalian manufacturing facility in US
News | November 25, 2024

Lonza announces first GMP batch completion at next-generation mammalian manufacturing facility in US

First GMP batch released at Lonza’s next-generation mammalian manufacturing facility in Portsmouth (US)


Azenta obtains regulatory approval for clinical long-read whole genome sequencing test
Biotech | November 22, 2024

Azenta obtains regulatory approval for clinical long-read whole genome sequencing test

Performed within GENEWIZ from Azenta Life Sciences' CLIA-certified and CAP-accredited state-of-the-art clinical genomics laboratory


USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency
Drug Approval | November 16, 2024

USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency

Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters


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