- PMLiVE

Pliant Therapeutics’ bexotegrast shows promise in rare liver disease patients

Primary sclerosing cholangitis affects more than 100,000 people worldwide

- PMLiVE

Researchers identify free light chain targets to help treat rare form of amyloidosis

AL amyloidosis is currently the most common and severe form of the condition

- PMLiVE

Chiesi’s rare disorder therapies approved by SMC for Fabry disease and epidermolysis bullosa

Rare diseases currently affect around 436,000 people living in Scotland

- PMLiVE

Roche announces CHMP recommendation for PiaSky in rare blood condition PNH

Paroxysmal nocturnal haemoglobinuria affects approximately 20,000 people worldwide

- PMLiVE

Savara announces positive phase 3 results for molgramostim in rare lung disease

Autoimmune pulmonary alveolar proteinosis affects around one in every 150,000 people in the US

- PMLiVE

FDA approves argenx’s Vyvgart Hytrulo for rare neuromuscular disorder CIDP

Approximately 24,000 US patients are currently being treated for chronic inflammatory demyelinating polyneuropathy

- PMLiVE

Takeda shares long-term results for HyQvia in rare neurological disorder CIDP

The therapy is the first and only facilitated subcutaneous immunoglobulin treatment for CIDP

- PMLiVE

Takeda shares late-stage results for investigational soticlestat in rare epileptic disorders

Dravet syndrome and Lennox-Gastaut syndrome are drug-resistant to many anti-seizure medications

- PMLiVE

Amgen’s Uplizna shows promise in rare fibroinflammatory disease IgG4-RD

The company is planning to file for US approval of the CD19-targeted drug in this patient population

- PMLiVE

Takeda’s enzyme replacement therapy recommended by CHMP for rare blood clotting disorder cTTP

If approved, rADAMTS13 will be the first and only enzyme replacement therapy in the EU indicated for cTTP

Biogen Idec building

Biogen’s Qalsody granted EC approval to treat rare form of ALS in adults

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

- PMLiVE

Amgen’s interchangeable Soliris biosimilar Bkemv approved by FDA for rare blood disorders

Bkemv has been authorised to treat paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome

- PMLiVE

Novartis shares positive late-stage results for Fabhalta in rare kidney disease C3 glomerulopathy

Approximately one to two people per million worldwide are diagnosed with C3G every year

Biogen Idec building

Biogen to expand immunology and rare disease portfolio with $1.8bn HI-Bio acquisition

The company will gain access to HI-Bio’s lead asset for immune-mediated and rare diseases

- PMLiVE

Pfizer’s Vyndaqel recommended by NICE to treat rare heart disease ATTR-CM

Approximately 1,500 people in England are affected by transthyretin amyloidosis cardiomyopathy