- PMLiVE

Researchers publish promising results for ultra-rare disease gene therapy method

Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency

- PMLiVE

Rare diseases: why they can be so difficult to identify

Symptoms shared with more common diagnoses can make it more difficult to spot underlying rare diseases

- PMLiVE

NICE recommends Ultomiris for NHS use in patients with rare blood disorders

aHUS is an ultra-rare and life-threatening disease that causes progressive injury to vital organs

- PMLiVE

Sarepta Therapeutics’ DMD gene therapy shows early success

Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens

- PMLiVE

BMS and bluebird bio gain FDA approval for multiple myeloma therapy

Abecma is a CAR T cell therapy approved for pre-treated myeloma patients

- PMLiVE

New data for Recordati’s Isturisa reinforces benefit in Cushing’s disease

Drug provided 'rapid and sustained' normalisation of mean urinary-free cortisol levels

- PMLiVE

bluebird bio reveals long-term data for Lenti-D gene therapy in CALD

At 24 months of follow-up, 90% of patients were free of major functional disabilities

- PMLiVE

bluebird bio says AML case “very unlikely” to be related to gene therapy LentiGlobin

bluebird bio's gene therapy LentiGlobin is used to treat sickle cell disease

- PMLiVE

Protalix, Chiesi’s monthly treatment PRX-102 shows benefit in Fabry disease

Fabry disease is a rare disorder in which patients inherit a deficiency of the α–Galactosidase–A enzyme

- PMLiVE

UK introduces Rare Diseases Framework to improve the lives of people with rare diseases

bluebird bio releases a video to raise awareness – Rare Disease Day 2021 is on 28 February

- PMLiVE

Rare genetic diseases – what’s in store for 2021?

Why personalised medicine based on our own genomes is the future of healthcare

- PMLiVE

Successful product commercialisation: what can we learn from the rare disease setting?

Achieving launch success in an increasingly challenging commercial environment

- PMLiVE

Chiesi launches new rare disease R&D division

New business unit will be based in Boston, US

Sanofi reception

Sanofi’s rare disease drug olipudase alfa scores in late-stage study

Met crucial endpoints across two clinical studies

- PMLiVE

Abeona Therapeutics’ rare disease phase 3 trial halted due to FDA concerns

Issues over transport stability must be resolved, says regulator