Patients, Providers & Caregivers
Uplifting life for people of all stripes.
© 2025 Zevra Therapeutics. All rights reserved. | Privacy Policy | Notice at Collection | Your Privacy Choices | Terms of Use
Niemann-Pick disease type C
Niemann-Pick disease type C (NPC) is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality.
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 can affect patients at any age. The symptoms for NPC are variable across patients, and many people with NPC experience a prolonged period before diagnosis due to the lack of disease awareness. Disease progression is irreversible in all patients and varies by the age of symptom onset.
Adult living with Niemann-Pick Disease Type C.
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. Ammonia is a waste product that your body makes when it digests protein.
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.
Vascular Ehlers-Danlos (VEDS)
Vascular Ehlers-Danlos (VEDS) syndrome is a severe autosomal, dominant, genetic, connective tissue disorder. Connective tissue holds all the body’s cells, organs, and tissue together.
VEDS is caused by mutations in the COL3A1 gene that tells the body how to make type Ill collagen, which is a major 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.
Vascular Ehlers-Danlos (VEDS)
Rare Sleep Disorders
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.
People living 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.
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.
People living 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.
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:
Expanded Access Policy
Working together to help people with rare diseases.