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Niemann Pick Type C
Niemann-Pick disease type C (NPC) is an ultra-rare, genetic, progressive and fatal neurological disease that presents itself in many different ways.
People with NPC face numerous, life-limiting challenges due to a malfunction of lysosomal storage and their body’s inability to appropriately transport and process cholesterol and lipids within their cells. As a result, NPC patients experience neurodegenerative effects due to the accumulation of lipids (fats) in the brain, spleen and liver.
NPC is an inherited disorder that can affect both children and adults. Disease progression is irreversible in all patients. Many people with NPC experience a prolonged period before diagnosis, followed by continued worsening of symptoms and disease progression over a decade or more, ultimately leading to death. Others may experience a more rapid decline.
We are developing arimoclomol for NPC.
We are developing KP1077 for rare sleep disorders, including IH.
Rare Sleep Disorders
Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness.
People with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep.
Additionally, those with IH have extreme difficulty waking, otherwise known as “sleep inertia,” severe “brain fog” and unintentionally falling asleep or at inappropriate times.
People with IH experience debilitating symptoms often further leading to reported memory problems, difficulty maintaining focus and depression.
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Working together to help people with rare diseases.