Rare diseases

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Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.

News

Electra sets terms for IPO, hoping to raise up to $347m

Electra Therapeutics has moved ahead with plans for an IPO, seeking to raise funds for its drug for the rare and life-threatening disease sHLH.

News

Reviews underway of first drug for rare disease MOGAD

Patients with rare autoimmune disease MOGAD could be months away from having a first approved therapy, as the FDA starts a review of Roche's Enspryng.

News

Pharvaris plans filings for HAE drug after pivotal trial win

Pharvaris has reported positive data from a phase 3 trial of its drug for preventing HAE attacks, setting up marketing applications next year.

News

Merck rare tumour drug Ezmekly backed for NHS use

Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.

News

Ionis claims first approval for Alexander disease therapy

Ionis' Zanvastro has been approved by the FDA for Alexander disease, becoming the first disease-modifying therapy for the devastating rare disease.

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Electra sets terms for IPO, hoping to raise up to $347m

Reviews underway of first drug for rare disease MOGAD

Pharvaris plans filings for HAE drug after pivotal trial win

Merck rare tumour drug Ezmekly backed for NHS use

Ionis claims first approval for Alexander disease therapy

Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

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Rare diseases

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