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Results For "rare-disease"

234 News Found

FDA grants orphan drug designation to MicuRx's MRX-5 for non-tuberculous mycobacteria infections
Drug Approval | January 02, 2025

FDA grants orphan drug designation to MicuRx's MRX-5 for non-tuberculous mycobacteria infections

MRX-5 is a novel benzoxazole antibiotic developed for the treatment of mycobacteria infections, particularly infections caused by non-tuberculous Mycobacteria


Immedica to acquire biopharmaceutical company Marinus Pharmaceuticals
News | December 31, 2024

Immedica to acquire biopharmaceutical company Marinus Pharmaceuticals

Immedica to commence a cash tender offer to acquire all issued and outstanding shares of Marinus for an enterprise value of approximately US$ 151 million


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


Pfizer appoints Chris Boshoff as Chief Scientific Officer and President, Research & Development
People | November 21, 2024

Pfizer appoints Chris Boshoff as Chief Scientific Officer and President, Research & Development

Roger Dansey to become Interim Chief Oncology Officer and Johanna Bendell to join Pfizer as Oncology Chief Development Officer


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


AstraZeneca Pharma India posts Q2 PAT at Rs. 38.43 Cr
News | November 14, 2024

AstraZeneca Pharma India posts Q2 PAT at Rs. 38.43 Cr

We are witnessing a steady and sustainable growth in the country as the Q2 revenue from operations increased by 31%


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


Fasenra approved in the US for eosinophilic granulomatosis with polyangiitis
Drug Approval | September 19, 2024

Fasenra approved in the US for eosinophilic granulomatosis with polyangiitis

New indication supported by the MANDARA trial which showed nearly 60% of patients achieved remission and 41% of patients fully stopped taking oral corticosteroids