- PMLiVE

Sanofi’s Xenpozyme not recommended by NICE for ultra-rare disease ASMD

An estimated 40 to 50 people in England are living with the lysosomal storage disorder

- PMLiVE

Sanofi’s Rezurock fast-tracked by NHS for rare chronic graft versus host disease

Around 400 stem cell or bone marrow transplant patients will develop the condition each year in England

- PMLiVE

FDA awards Orphan Drug Designation to SynaptixBio for rare brain disease therapy

Isolated hypomyelination is a form of TUBB4A leukodystrophy that has been identified relatively recently

- PMLiVE

Radboud researchers use new method to improve diagnosis of rare diseases

Approximately 7,000 rare diseases collectively impact up to 5.9% of the global population

- PMLiVE

OneChain-led consortium granted €1.9m to advance CAR-T therapy for rare leukaemia

Acute lymphoblastic leukaemia has an incidence of around 1.5 cases per 100,000 people

- PMLiVE

Clinigen launches ‘What is Possible?’ campaign on Rare Disease Day to accelerate access to medicines

There are over 7,000 rare diseases impacting more than 300 million people globally

- PMLiVE

CHMP recommends Travere and CSL Vifor’s Filspari to treat rare kidney disease IgAN

The rare form of kidney disease is estimated to affect around 25 per million adults worldwide

- PMLiVE

Rare diseases – driving further improvements in patient access

Despite significant strides, many rare disease patients have no licensed treatment options and face high levels of unmet need

- PMLiVE

AstraZeneca’s Voydeya receives CHMP recommendation for rare blood disease PNH

Up to 20% of PNH patients treated with a C5 inhibitor experience clinically significant extravascular haemolysis

Biogen Idec building

Biogen’s Qalsody recommended by CHMP to treat rare form of ALS

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

- PMLiVE

Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

- PMLiVE

NICE recommends STADA and Calliditas’ Kinpeygo to treat rare kidney disease IgAN

Affecting approximately 14,000 in England, IgA nephropathy occurs when IgA accumulates in the kidneys, causing inflammation and scarring

- PMLiVE

Researchers identify five rare cases of Alzheimer’s from historic medical treatments

The study found that c-hGH treatment transmitted the amyloid-beta protein, a hallmark of AD

- PMLiVE

Takeda’s HyQvia receives EC approval for rare neuromuscular disorder CIDP

HyQvia can now be used as a maintenance therapy for patients of all ages with CIDP

- PMLiVE

Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency