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Niemann-Pick disease type C
Niemann-Pick disease type C (NPC) is an ultra-rare, genetic neurological disease that presents itself in many different ways.
People with NPC have changes in their lysosomal function impacting their body’s ability to appropriately transport cholesterol and other lipids within their cells. Over time, this accumulation causes progressive dysfunction of the nerves, brain, and other organs, resulting in neurodegenerative and visceral symptoms. These symptoms include negative impacts on speech, cognitive function, fine motor skills, and the ability to swallow.
NPC is an inherited disorder that has a highly heterogeneous and variable symptom presentation across patients. Many people with NPC experience a prolonged period before diagnosis due to the lack of awareness of NPC. Disease progression is irreversible in all patients and varies by the age of symptom onset. NPC is a life limiting disease and causes premature mortality.
Ara Parseghian Medical Research Fund – Fighting for Cure for Niemann-Pick Type C Disease
Hope for Marian
International Niemann–Pick Disease Alliance
International Niemann-Pick Disease Registry
National Niemann-Pick Disease Foundation
Niemann-Pick UK
Shining a Light on NPC – Firefly Fund
Zevra’s Information on Arimoclomol
We are developing arimoclomol for NPC.
Urea Cycle Disorders.
Urea Cycle Disorders
Urea cycle disorders (UCDs) are rare genetic metabolic disorders that impair the body’s ability to remove excess ammonia.
People living with UCDs often suffer from ammonia buildup in the body, potentially causing brain damage, neurocognitive impairments, liver disease, coma and even death. It is important that patients adhere to lifelong treatment, which may include a protein restricted diet and medications such as nitrogen scavengers, to control ammonia levels.
Rare Sleep Disorders
Learn more about rare sleep disorders Idiopathic Hypersomnia and Narcolepsy
About Idiopathic Hypersomnia
Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness. Patients 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 often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression.
It is estimated that approximately 37,000 patients in the United States are currently diagnosed with IH and seeking treatment, although the total patient population may be much larger due to some patients not seeking treatment or being undiagnosed or misdiagnosed.
Narcolepsy is a chronic debilitating central disorder of hypersomnolence. The primary symptom of narcolepsy is excessive daytime sleepiness characterized by daily episodes of an irrepressible need to sleep or daytime lapses into sleep. Patients with narcolepsy have an abnormal rapid eye movement (REM) sleep phase which can cause disrupted nighttime sleep, sleep paralysis and sleep-related hallucinations during sleep-wake transitions. Narcolepsy has severe personal, social, and economic consequences. Patients with narcolepsy experience substantial impairment of their mental and physical wellbeing, and depression and anxiety are common. Cognitive dysfunctions such as difficulty to focus and memory lapses (also referred to as ‘brain fog’) are frequently reported. The many symptoms experienced by patients with narcolepsy result in a high disease burden and poor quality of life.
Narcolepsy is categorized in to two types: narcolepsy type 1 (NT1) and type 2 (NT2). NT1 is considered a distinct disease entity characterized in part by loss of hypocretin neurons and symptoms of cataplexy (sudden, brief attacks of muscle weakness sometimes resulting in the body to fall uncontrollably, often triggered by strong emotions). When narcolepsy presents without cataplexy and with normal hypocretin-1 concentrations in the cerebrospinal fluid (CSF), it is categorized as NT2 (Hypocretin-1 is also known as orexin-A, a neuropeptide involved in regulating sleep-wake cycles).
The combined worldwide prevalence of both types of narcolepsy has been estimated to be 25-50 per 100,000 people. Epidemiological studies using well-defined criteria for assessing the prevalence of narcolepsy (both NT1 and NT2) estimate incidence rates ranging from 31 to 79 per 100,000 people corresponding to approximately 100,000 to 260,000 total patients in the United States.
We are developing KP1077 for rare sleep disorders, including IH.
We are developing Celiprolol for rare Vascular Ehlers-Danlos (VEDS).
Vascular Ehlers-Danlos (VEDS)
Vascular Ehlers-Danlos (VEDS) syndrome is a severe autosomal dominant genetic connective, tissue disorder characterized by risk of dissection and rupture of the arteries, gastrointestinal tract, and uterus.
VEDS is caused by mutations in the COL3A1 gene that encodes type Ill collagen, which is a major extracellular matrix component of the vasculature and hollow organs. People living with VEDS often experience frequent and unexplained bruising, and thin and translucent skin.
Most people living with VEDS will experience severe life-altering, and life-threatening complications such as gastrointestinal perforations, spontaneous aorta or arterial tears, dissections or ruptures, and spontaneous pneumothorax, all of which can be fatal. Median life expectancy for VEDS patients is 51 years of age, with the first severe vascular events occurring in the early 20’s.
It is estimated that approximately 7,500 patients in the United States are currently diagnosed with VEDS and seeking treatment. However, the total patient population may be larger due to some patients not seeking treatment or being undiagnosed or misdiagnosed.
KP1077.D01
A Clinical Study in Adults with Idiopathic Hypersomnia (IH)
NOW ENROLLING adults (18 years and older) with IH
Study KP1077.D01 is being conducted by Zevra to evaluate the safety and efficacy of KP1077 capsules in patients with IH.
KP1077 capsules contain serdexmethylphenidate (SDX), a prodrug of dexmethylphenidate. It is an investigational medication for treating excessive daytime sleepiness (EDS) and other symptoms of IH, including sleep inertia (difficulty of waking up in the morning), and brain fog (lack of focus and mental clarity; forgetfulness and confusion). SDX has a unique, slow release profile that could potentially provide stable control of sleepiness throughout the day, with low abuse potential.
The clinical study consists of a screening period (up to 5 weeks), a 5-week open-label dose optimization period during which the optimal SDX dose will be determined, followed by a 2-week double-blind, randomized withdrawal period.
For more information or to find a participating clinical site:
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