Neuren receives rare paediatric disease designation from the US FDA

February 24, 2025 Australian Biotech

This is a preview of subscriber content

Subscribe now to continue reading.

Australian company Neuren Pharmaceuticals has received a Rare Paediatric Disease Designation from the US FDA for NNZ-2591 in Pitt-Hopkins syndrome and Angelman syndrome. …

Subscribe now to continue reading.

Latest Video

New Stories

Medicines Australia chief says next negotiation is about getting comfortable with being uncomfortable
July 3, 2026 - - Latest News
New once weekly therapy offers extended protection for people with haemophilia A
July 3, 2026 - - Latest News
Nurse prescribing reform set to expand access to PBS subsidised medicines
July 3, 2026 - - Latest News
Nothing else grows below this self-serving and broken institutional framework
July 3, 2026 - - Latest News
Minister looks to be sending a very clear message about these therapies and the PBS
July 2, 2026 - - Latest News
Cartherics emerges as Asia Pacific leader in off-the-shelf immune cell therapy
July 2, 2026 - - Australian Biotech
The end-game of strong-arming companies over prices at the expense of patients and process
July 2, 2026 - - Latest News

Neuren receives rare paediatric disease designation from the US FDA

This is a preview of subscriber content

Latest Video

Most Read

Patient alarm over the potential loss of subsidised access to MS medicines

Minister looks to be sending a very clear message about these therapies and the PBS

The minister is following a tried and tested process, but what is the risk in this new world?

Major new survey shows patients paying more and waiting longer for medicines

Listing delivers access to targeted therapy for Australians living with myasthenia gravis

New Stories

Medicines Australia chief says next negotiation is about getting comfortable with being uncomfortable

New once weekly therapy offers extended protection for people with haemophilia A

Nurse prescribing reform set to expand access to PBS subsidised medicines

Nothing else grows below this self-serving and broken institutional framework

Minister looks to be sending a very clear message about these therapies and the PBS

Cartherics emerges as Asia Pacific leader in off-the-shelf immune cell therapy

The end-game of strong-arming companies over prices at the expense of patients and process

Sign In

Neuren receives rare paediatric disease designation from the US FDA

This is a preview of subscriber content

Latest Video

Most Read

Patient alarm over the potential loss of subsidised access to MS medicines

Minister looks to be sending a very clear message about these therapies and the PBS

The minister is following a tried and tested process, but what is the risk in this new world?

Major new survey shows patients paying more and waiting longer for medicines

Listing delivers access to targeted therapy for Australians living with myasthenia gravis

Follow Us

New Stories

Medicines Australia chief says next negotiation is about getting comfortable with being uncomfortable

New once weekly therapy offers extended protection for people with haemophilia A

Nurse prescribing reform set to expand access to PBS subsidised medicines

Nothing else grows below this self-serving and broken institutional framework

Minister looks to be sending a very clear message about these therapies and the PBS

Cartherics emerges as Asia Pacific leader in off-the-shelf immune cell therapy

The end-game of strong-arming companies over prices at the expense of patients and process