- PMLiVE

Researchers uncover role of inherited genetic variants in rare blood cancer

There are around 4,000 cases of myeloproliferative neoplasms in the UK each year

- PMLiVE

Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

- PMLiVE

AstraZeneca and Ionis’ Wainua granted FDA approval for rare disease ATTRv-PN

ATTRv-PN leads to peripheral nerve damage with motor disability within five years of diagnosis

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EC approves Blueprint’s Ayvakyt as first treatment for rare haematological disorder

The therapy targets the primary underlying cause of indolent systemic mastocytosis

- PMLiVE

Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

The rare blood disease is estimated to affect only ten to 20 people per million worldwide

- PMLiVE

NHS to offer stem cell transplants to patients living with rare blood disorder

Over 600 adults in the UK are affected by transfusion-dependant thalassaemia

- PMLiVE

AstraZeneca’s rare disease unit receives NICE recommendation for Wolman disease therapy in infants

The enzyme replacement therapy will become the first treatment available on the NHS for the rare genetic disease

- PMLiVE

Takeda’s enzyme replacement therapy approved by FDA for rare blood clotting disorder

The ultra-rare inherited disease is estimated to affect fewer than 1,000 people in the US

- PMLiVE

Novartis shares positive phase 3 results for atrasentan in rare kidney disease

The company gained access to the candidate after it acquired Chinook Therapeutics

- PMLiVE

FDA approves Servier’s Tibsovo as first targeted therapy for rare blood cancer

Approximately 16,000 people in the US are diagnosed with myelodysplastic syndromes every year

- PMLiVE

Neuromyelitis optica spectrum disorder: is there hope on the horizon?

The new science behind the management of this devastating rare autoimmune disease

- PMLiVE

LifeArc to launch rare disease centres across the UK

One in 17 people will be affected by a rare disease at some point in their lives, amounting to around 3.5 million people in the UK

- PMLiVE

Landscape review reveals £1.1bn of UK funding for rare diseases

The report focuses on future funding and needs for those with rare diseases

- PMLiVE

Clinigen launches educational programme for rare disease patients and organisations

The NaviGATE programme will aim to empower patients and provide navigational support

- PMLiVE

AstraZeneca’s rare disease unit partners with Verge Genomics in deal worth over $840m

Both will identify drug targets for neurodegenerative and neuromuscular diseases