Applying
Brave
Science to Diseases
and Conditions
We have developed FDA-approved therapies for tardive dyskinesia and Parkinson’s disease and are working on developing potential treatments across multiple disease states.
from complex disease
states, our team
invested in bringing medicines
to market for people with under-
addressed and rare diseases.
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TD is associated with taking certain kinds of mental health medicines (antipsychotics) commonly prescribed to treat mental illnesses such as depression, bipolar disorder, schizophrenia, and schizoaffective disorder. Treatment with antipsychotics are thought to result in irregular dopamine signaling in a region of the brain that controls movement. TD is estimated to affect approximately 600,000 people in the U.S.
PD affects approximately 1 million people in the U.S. and 6 million people worldwide. PD is caused by low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking, and writing. As PD progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing. PD may also include non-motor symptoms, such as fatigue/sleep disorders, memory problems, and weight change, among others.
There is presently no cure for PD, and management of the disease consists of the use of treatments that attempt to control motor symptoms primarily through dopaminergic mechanisms. The current gold standard for treatment of motor symptoms is levodopa/carbidopa. While levodopa/carbidopa improves patients’ motor symptoms, as the disease progresses, the effects of levodopa begin to wear off more quickly. Patients then experience motor fluctuations throughout the day between ON time, periods when the medication is working and PD symptoms are controlled, and OFF time, when the medication is not working and PD symptoms return.
HD is a hereditary, progressive neurodegenerative disorder in which neurons within the brain break down. HD can cause movement, cognitive, and psychiatric disorders with a wide range of signs and symptoms. Symptoms generally appear between ages 30 to 50 and worsen over a 10- to 25-year period. Many people with HD experience chorea, a troublesome involuntary movement disorder, in which patients develop abnormal, abrupt, or irregular movements.
Roughly 90% of the approximately 41,000 patients in the U.S. diagnosed with HD will develop chorea over the course of the disease.
Approximately 15% of the estimated 600,000 to 1 million people living in the U.S. diagnosed with cerebral palsy live with dyskinesia (uncontrollable muscle movements).
There are no approved treatments for DCP.
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EE-CSWS typical onset occurs between the ages of two and four years old, with seizures occurring primarily during sleep. The seizures interfere with processes critical to learning and memory. Diagnosis of EE-CSWS is based on a unique electroencephalogram (EEG) pattern of electrical status epilepticus during sleep (ESES), together with cognitive stagnation and regression. Following puberty, seizure frequency tends to decline, however, developmental delays often remain.
EE-CSWS impacts less than 2% of children living with epilepsy worldwide. There is currently no approved treatment for the disorder.
Focal onset seizures can last up to several minutes, and can involve involuntary movements with alteration or loss of awareness. These seizures can interfere with activities of daily living and negatively impact quality of life.
Over half of the 45 million people who live with active epilepsy worldwide have focal onset seizures.
Watch an Expert on Focal Onset Seizures Explain Symptoms and Current Treatments
Resource
SCN8A-DEE causes a range of symptoms including severe epilepsy, early onset developmental delay, cognitive impairment, and other medical challenges. Typical onset occurs around 4 months of age.
There are no approved therapies for this form of pediatric epilepsy.
Hear From Gabi, a Caregiver to a Child With SCNA8-DEE
Continue to Learn More About Gabi’s Story >
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Congenital Adrenal Hyperplasia (CAH)
CAH is a group of rare autosomal recessive disorders that result in an enzyme deficiency (~95% due to 21-hydroxylase [21-OHD] deficiency) that alters the production of adrenal hormones.
If left untreated, classic CAH, the more severe form of the disorder, can result in salt wasting, dehydration, and even death. Both males and females with classic CAH can experience problems with growth and development in childhood, including early puberty, short stature, or height below genetic potential, and fertility problems in adulthood. Classic CAH due to 21-OHD deficiency is estimated to affect up to approximately 30,000 people in the U.S. and 50,000 people in Europe.
There are no non-steroidal treatments approved by the U.S. Food and Drug Administration for classic CAH. Glucocorticoids are the current standard of care for people living with the disorder and are used to correct the body’s cortisol deficiency and attempt to reduce the high adrenocorticotropic hormone (ACTH) levels in an attempt to control androgen excess.
However, the dose and duration of glucocorticoids required to suppress ACTH in order to control androgen levels in people living with classic CAH are often well above what are needed for replacement dosing to treat the cortisol deficiency, which can result in serious and common complications of steroid excess, including metabolic abnormalities, bone loss, growth impairment, increased risk of infections, and increased risk of cardiovascular disease.
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As one of the leading causes of disability worldwide, schizophrenia often results in significant emotional burden for those who experience symptoms, as well as their family and friends. It impacts approximately 3.5 million people in the U.S. alone and more than 20 million people worldwide. Approximately 30% of patients with schizophrenia fail to respond to current antipsychotic therapy.
MDD is characterized by a persistently depressed mood, loss of interest, lack of enjoyment in daily activities, and decreased energy that can impact normal daily functioning, relationships, and overall quality of life. Of the more than 16 million people in the U.S. who live with MDD, about one-third do not respond to available antidepressants.
Anhedonia is a core symptom of major depressive disorder (MDD), and frequently occurs in people with schizophrenia, bipolar disorder, substance abuse, Parkinson’s disease, diabetes, and coronary artery disease. Anhedonia has been associated with changes in neurotransmitter levels involved in the brain’s reward system.
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Symptoms of endometriosis include painful periods, pelvic pain between periods, and pain with sex. Estrogen fuels the growth of lesions that can occur on the ovaries, the fallopian tubes, or other areas near the uterus, such as the bowel or bladder.
According to researchers, 7.5 million women in the U.S. are diagnosed with endometriosis, with 3 million diagnosed with moderate to severe endometriosis.
*All commercialization and marketing by AbbVie Inc.
Uterine fibroids are most often seen in women between ages 30 to 40 but can occur at any age. Uterine fibroids are the most common pelvic growth, affecting around 20% of all women by age 59. Uterine fibroids are the leading cause of infertility.
Some common symptoms associated with uterine fibroids include heavy menstrual bleeding, painful periods, vaginal bleeding at times other than menstruation, anemia, pain in the abdomen or lower back, pain during sex, difficulty urinating or frequent urination, constipation, rectal pain, or difficulty getting pregnant.
*All commercialization and marketing by AbbVie Inc.
References
2. MedlinePlus. Movement disorders. Accessed July 12, 2022. https://medlineplus.gov/movementdisorders.html
3. Epilepsy Foundation. Who can get epilepsy? Accessed July 12, 2022. https://www.epilepsy.com/learn/about-epilepsy-basics/who-gets-epilepsy
4. National Organization for Rare Disorders. Congenital adrenal hyperplasia. Accessed July 12, 2022. https://rarediseases.org/rare-diseases/congenital-adrenal-hyperplasia/
5. Our World in Data. Mental health. Accessed July 12, 2022. https://ourworldindata.org/mental-health
6. National Library of Medicine. MedlinePlus. Uterine fibroids. Accessed July 12, 2022. https://medlineplus.gov/uterinefibroids.html#
7. National Library of Medicine. MedlinePlus. Endometriosis. Accessed July 12, 2022. https://medlineplus.gov/endometriosis.html
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