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Novartis’ Kisqali receives CHMP recommendation for early breast cancer patients

Approximately 70% of all breast cancer patients have HR-positive/HER2-negative disease

- PMLiVE

FDA advisory committee recommends Stealth’s elamipretide for Barth syndrome

The ultra-rare genetic disease affects approximately 150 people in the US

- PMLiVE

Roche presents two-year data for spinal muscular atrophy treatment Evrysdi in children

The severe and progressive neuromuscular disease affects about one in every 10,000 babies

- PMLiVE

NHS England begins newborn screening programme to identify rare genetic conditions

The Generation Study is aiming to screen 100,000 newborns for more than 200 diseases

- PMLiVE

FDA approves IntraBio’s Aqneursa to treat Niemann-Pick disease type C patients

The rare lysosomal storage disorder affects approximately one in every 100,000 to 120,000 births

- PMLiVE

MHRA approves Pharming’s Joenja as first drug for rare immune disease APDS

Up to 40 people in the UK are affected by activated phosphoinositide 3-kinase delta syndrome

- PMLiVE

FDA grants rare paediatric disease designation to Enterprise’s cystic fibrosis candidate

The genetic disease is estimated to affect more than 100,000 people globally

- PMLiVE

Championing people-powered communications in rare diseases: AMICULUM’s feature in The Guardian Rare Diseases Campaign 2024

By Ellie Thomas

Amiculum

- PMLiVE

Novo Nordisk and NanoVation to develop genetic medicines in partnership worth $600m

The companies will collaborate on up to seven programmes for cardiometabolic and rare diseases

- PMLiVE

Novartis’ Kisqali combination granted FDA approval for early breast cancer patients

The drug is already approved for certain cases of advanced or metastatic breast cancer

Biogen Idec building

Biogen shares promising results for higher dose nusinersen in spinal muscular atrophy

The neuromuscular disease affects approximately one in every 10,000 babies worldwide

- PMLiVE

Novartis’ Fabhalta recommended by NICE to treat rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately ten to 20 people per million worldwide

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