The National Urea Cycle Disorders Foundation (NUCDF) and Zevra
Therapeutics, Inc. (NasdaqGS: ZVRA) are teaming up to launch an
awareness campaign during the month of October to improve the
diagnosis of urea cycle disorders (UCDs). Their goal is to
highlight the critical importance of promptly checking blood
ammonia levels and using proper testing techniques.
A blood ammonia test is essential to uncover
elevated ammonia levels (hyperammonemia), the classic sign of a
urea cycle disorder. Resources on recognizing and testing for
hyperammonemia are featured on the campaign website,
checkammonia.com.
Quickly diagnosing UCDs is critical. Toxic
ammonia levels can rise rapidly in affected individuals, which if
left untreated, can cause coma and death.
Newborns with severe UCDs typically become
catastrophically ill within 36-48 hours after birth. It is
estimated that up to 20% of sudden infant death syndrome (SIDS)
cases may be attributed to an undiagnosed inborn error of
metabolism such as a UCD. Adults with undiagnosed UCDs may present
at emergency rooms with staggering, confusion, combativeness and
disorientation that is mistaken for alcohol or drug intoxication,
and many die.
The campaign’s primary message is that a blood
ammonia test, performed promptly and properly, can save lives.
Downloadable campaign resources focus on (1) Recognizing the signs
of UCDs and promptly ordering a test and (2) Following proper test
procedures.
About Urea Cycle Disorders
A urea cycle disorder is caused by a genetic
mutation—a change in a person’s genes—that leads to a deficiency of
one of the enzymes needed to metabolize protein. These missing or
reduced enzymes make the process less efficient, allowing ammonia
to build up in the bloodstream. High ammonia levels are toxic to
the brain, causing a range of physical, neurological, and
behavioral symptoms.
UCDs can strike at any age. Those with more
severe UCDs (greater enzyme deficiency) typically become
symptomatic very shortly after birth. Those with less severe UCDs
(less enzyme deficiency) can develop symptoms at any time later in
life.
Signs of UCDs
The Check Ammonia campaign website highlights
the early signs of hyperammonemia in newborns and in infants,
children, and adults that should prompt a blood ammonia test. Key
warning signs include:
-
Newborns from 1 to 28 days old: poor feeding or
vomiting, hypotonia (poor muscle tone or floppiness), lethargy
(difficult to wake up), respiratory distress, seizures, or
coma.
- Older
infants and children: inconsolable crying, agitation, or
hyperactive behavior sometimes accompanied by screaming,
self-injurious behavior, and refusal to eat meat or other
high-protein foods. Later symptoms may include frequent episodes of
vomiting, especially following high-protein meals, lethargy and
delirium. Older children may be referred to child psychologists
because of their behavior, developmental delays, and eating
problems.
- Adults
with high blood ammonia levels: disorientation, confusion,
slurred speech, unusual and extreme combativeness or agitation,
stroke-like symptoms, lethargy and delirium. Many may be seen
by neurologists or psychiatrists because of psychiatric symptoms,
including schizophrenia and bipolar disorder.
Who should get tested?
Routine ammonia checks aren’t necessary for
everyone, but the test is crucial for those experiencing symptoms
that could be high ammonia.
How is high ammonia
treated?
Once identified, high ammonia levels can be
managed and treated in several ways. These treatments may include
medications, dietary changes including protein restriction,
managing underlying conditions, or hospitalization in more severe
cases, especially if neurological symptoms are present.
Join the Campaign
Patients, providers, and the public are invited
to help share the campaign’s messages and materials widely. For
electronic copies of campaign materials, visit checkammonia.com.
Contact info@nucdf.org with questions or to request hard copies you
can deliver to pediatricians, hospitals, emergency rooms, and other
healthcare providers in your area. Follow the NUCDF’s social media
sites (Facebook, LinkedIn, Instagram and YouTube) to share
materials throughout the month.
Learn more and hear patient
stories:
Watch a video on recognizing the signs of urea
cycle disorders.
Read about Jennings, an infant who passed away
three days after he was born due to an undiagnosed UCD.
Hear about Zoey, a 19-year-old girl who lost her
life suddenly at age 19 from an undiagnosed urea cycle
disorder.
Read about Jim Stavas, a fireman who fell into a
coma and died after sinus surgery due to an undiagnosed UCD (under
“Why we ride”).
About the National Urea Cycle Disorders
Foundation: NUCDF is a nonprofit organization dedicated to
saving and improving the lives of children and adults affected by
urea cycle disorders (UCDs) and raising awareness since 1988. Led
exclusively by patients and families affected by UCDs, we are the
driving force behind critical research to improve the understanding
and management of UCDs, find new treatments, and ultimately a cure.
NUCDF serves as a lifeline to patients, families, and medical
professionals worldwide seeking information, support, and hope.
Visit www.nucdf.org or email info@nucdf.org.
About Zevra Therapeutics:
Zevra Therapeutics, Inc. is a commercial-stage
rare disease company combining science, data, and patient needs to
create transformational therapies for diseases with limited or no
treatment options. Our mission is to bring life-changing
therapeutics to people living with rare diseases. With unique,
data-driven development and commercialization strategies, the
Company is overcoming complex drug development challenges to make
new therapies available to the rare disease community.
For more information, please visit www.zevra.com
or follow us on X (formerly Twitter) and LinkedIn.
Caution Concerning Forward-Looking
Statements:
This press release may contain forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements include all
statements that do not relate solely to historical or current
facts, including without limitation statements regarding upcoming
events or Zevra’s participation at such events. Forward-looking
statements are based on information currently available to Zevra
and its current plans or expectations. They are subject to several
known and unknown uncertainties, risks, and other important factors
that may cause our actual results, performance, or achievements to
be materially different from any future results, performance, or
achievements expressed or implied by the forward-looking
statements. These and other important factors are described in
detail in the "Risk Factors" section of Zevra’s Annual Report on
Form 10-K for the year ended December 31, 2023, Zevra’s Quarterly
Report for the quarter ended June 30, 2024, and Zevra’s other
filings with the Securities and Exchange Commission. While we may
elect to update such forward-looking3 statements at some point in
the future, except as required by law, we disclaim any obligation
to do so, even if subsequent events cause our views to change.
Although we believe the expectations reflected in such
forward-looking statements are reasonable, we cannot assure that
such expectations will prove correct. These forward-looking
statements should not be relied upon as representing our views as
of any date after the date of this press release.
NUCDF Contact
Jill Williams, 513-288-1509,
jwilliams@nucdf.org
Zevra Contact
Nichol Ochsner, +1 (732) 754-2545,
nochsner@zevra.com
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