Pompe disease

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Astellas licenses companion drug for Pompe gene therapy

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe

News

CHMP backs Amicus' therapy for Pompe disease

Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines

News

Another Astellas gene therapy hits a safety hurdle

The safety of Astellas' gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845.

News

Astellas says Pompe gene therapy clears safety hurdle

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate

News

Sanofi extends its Pompe range with first Nexviazyme OK

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder.

News

Boost for AZ as Ultomiris gets CHMP nod for expanded label

Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion of the label of Ultomiris, one of the main assets behind the

Astellas licenses companion drug for Pompe gene therapy

CHMP backs Amicus' therapy for Pompe disease

Another Astellas gene therapy hits a safety hurdle

Astellas says Pompe gene therapy clears safety hurdle

Sanofi extends its Pompe range with first Nexviazyme OK

Boost for AZ as Ultomiris gets CHMP nod for expanded label

Clinical trial go-between Inato raises $20m

Trials and tribulations of drug development

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