Touting 100% OS rate in pivotal rare disease trial, Rocket Pharma ready to head to FDA

Amber Tong

In drug development, you don’t throw around the word “cure” lightly. But Rocket Pharmaceuticals’ latest cut of gene therapy data has at least one analyst …

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

BECOME A PREMIUM SUBSCRIBER

Touting 100% OS rate in pivotal rare disease trial, Rocket Pharma ready to head to FDA

Amber Tong

Sign up to read this article for free.

Updated: Amgen obesity shot shows 20% weight loss, but dosing questions hang over readout

The Biden administration wants Medicare and Medicaid to cover GLP-1s. Will the Trump White House allow it?

Sarepta licenses Arrowhead's RNA-based rare disease pipeline, promising big biobucks

Axsome moves toward NDA filing for narcolepsy drug following positive Phase 3 data

Soleno’s rare pediatric disease drug faces PDUFA delay by three months

Agilent reveals plans to remodel operations as annual revenue dips

Tout­ing 100% OS rate in piv­otal rare dis­ease tri­al, Rock­et Phar­ma ready to head to FDA

Amber Tong

Sign up to read this article for free.

more like this

Up­dat­ed: Am­gen obe­si­ty shot shows 20% weight loss, but dos­ing ques­tions hang over read­out

The Biden ad­min­is­tra­tion wants Medicare and Med­ic­aid to cov­er GLP-1s. Will the Trump White House al­low it?

Sarep­ta li­cens­es Ar­row­head­'s RNA-based rare dis­ease pipeline, promis­ing big biobucks

Ax­some moves to­ward NDA fil­ing for nar­colep­sy drug fol­low­ing pos­i­tive Phase 3 da­ta

Soleno’s rare pe­di­atric dis­ease drug faces PDU­FA de­lay by three months

Ag­i­lent re­veals plans to re­mod­el op­er­a­tions as an­nu­al rev­enue dips

Touting 100% OS rate in pivotal rare disease trial, Rocket Pharma ready to head to FDA

Updated: Amgen obesity shot shows 20% weight loss, but dosing questions hang over readout

The Biden administration wants Medicare and Medicaid to cover GLP-1s. Will the Trump White House allow it?

Sarepta licenses Arrowhead's RNA-based rare disease pipeline, promising big biobucks

Axsome moves toward NDA filing for narcolepsy drug following positive Phase 3 data

Soleno’s rare pediatric disease drug faces PDUFA delay by three months

Agilent reveals plans to remodel operations as annual revenue dips