Venglustat is in clinical development for the treatment of patients with Gaucher disease type 3 (GD3) who have been effectively treated with Enzyme Replacement Therapy (ERT) for at least 3 years. Gaucher Disease (GD) is a genetic disorder caused by an enzyme deficiency where fatty substances build up in areas such as the spleen, liver, […]
PXT3003 is currently in clinical development for treatment of patients aged 16-65 years with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT is a group of inherited conditions that damage the peripheral nerves (reside outside the brain and spinal cord). CMT1 is characterised by muscle weakness and atrophy, which can lead to repeated ankle sprains, and changes […]
Pirtobrutinib is currently in clinical development for the treatment of patients with chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) who have previously received treatment with the current standard of care. CLL and SLL are types of cancer in which too many white blood cells (B-cells) are produced, these cells build up in the […]
Galcanezumab is in clinical development for the preventative treatment of paediatric patients with episodic or chronic migraine. Migraine is one of the most common neurological conditions in the paediatric population with unique clinical characteristics that can evolve with age. These characteristics can include shorter attacks, different location of pain (bilateral in younger children), and light […]
Pegcetacoplan is in clinical development for the treatment of patients with amyotrophic lateral sclerosis (ALS), a form of motor neurone disease. ALS is a progressive disease where the nerve cells responsible for sending instructions to the muscles gradually deteriorate, leading to weakness, muscle wasting and paralysis. ALS is a debilitating and life-threatening disease. Currently, there […]
Ravulizumab is in development for the treatment of thrombotic microangiopathies following haematopoietic stem cell transplantation (HSCT-TMA) in children, adolescents, and adults. Thrombotic microangiopathies (TMA) are clinical syndromes defined by the destruction of red blood cells, low platelets, and organ damage due to the formation of microscopic blood clots in capillaries and small arteries. Haematopoietic stem […]
Tiragolumab in combination with atezolizumab is in clinical development to treat patients withunresectable oesophageal squamous cell carcinoma (ESCC) following chemoradiotherapy. ESCCis a type of cancer that begins in the thin, flat cells lining the food pipe (oesophagus) and mayspread to other parts of the body. Unresectable refers to cancer which cannot be removedcompletely by surgery. […]
Fosdenopterin is currently in clinical development for the treatment of paediatric patients aged 1 day to 5 years with molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP). MoCD type A is a rare genetic disease that can appear shortly after birth and […]
Pembrolizumab in combination with Bacillus Calmette-Guerin (BCG) is in development for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC) that is recurrent or persistent following BCG induction or naïve to BCG treatment. Bladder cancer is when cancerous cells develop in the lining of the bladder. NMIBC is the most common type of bladder cancer […]
Cabotegravir is in clinical development as an alternative method of protecting an individual from contracting the human immunodeficiency virus type 1 (HIV-1). HIV is a chronic life altering condition that requires, daily antiretroviral therapy (ART) as a regular treatment routine. If left unmanaged it can lead to serious complications, such as acquired immunodeficiency syndrome (AIDS). […]
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