- PMLiVE

DEBRA and Queen Mary partner to advance epidermolysis bullosa drug development

Around 500,000 people worldwide are affected by the group of rare genetic skin conditions

- PMLiVE

FDA approves Jazz Pharmaceuticals’ Modeyso as first drug for ultra-rare brain cancer

H3 K27M-mutant diffuse midline glioma affects an estimated 2,000 people in the US every year

- PMLiVE

FDA approves Apellis’ Empaveli for two rare kidney diseases

C3G and primary IC-MPGN are estimated to affect 5,000 people in the US

- PMLiVE

FDA approves PTC Therapeutics’ Sephience to treat rare metabolic disorder PKU

Phenylketonuria affects one in every 13,500 to 19,000 newborns in the US

- PMLiVE

Novo Nordisk’s Alhemo approved by FDA for expanded haemophilia use

The rare bleeding disorder affects approximately 800,000 people globally

- PMLiVE

Roche’s Evrysdi tablets approved by MHRA to treat spinal muscular atrophy

It is hoped the new formulation will offer patients greater freedom and flexibility compared to the liquid version

Biogen Idec building

Biogen’s Qalsody granted MHRA approval to treat rare form of motor neurone disease

Mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease

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EC approves SpringWorks Therapeutics’ Ezmekly to treat rare genetic disorder NF1

Neurofibromatosis type 1 affects an estimated 135,000 people in the EU

- PMLiVE

NICE recommends BeOne’s Brukinsa to treat mantle cell lymphoma on NHS

Approximately 600 people are affected by the form of non-Hodgkin lymphoma every year in the UK

- PMLiVE

Alnylam’s Amvuttra approved by MHRA to treat rare heart disease ATTR-CM

The drug has been authorised to treat both wild-type and hereditary forms of the disease

- PMLiVE

MHRA approves KalVista Pharmaceuticals’ Ekterly to treat hereditary angioedema attacks

Ekterly is now the first and only oral on-demand treatment for HAE to be approved by the UK regulator

- PMLiVE

Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE

The rare inherited disease affects approximately 11,000 people in the UK

- PMLiVE

Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval

The rare inherited disease affects more than 109,000 people worldwide

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EC approves Jazz Pharmaceuticals’ Ziihera for advanced biliary tract cancer

Approximately 26% of biliary tract cancer patients globally have HER2-positive disease

- PMLiVE

UCB shares promising results for fenfluramine in ultra-rare form of epilepsy

CDKL5 deficiency disorder occurs in approximately one in 40,000 to 60,000 live births