NUCDF and Zevra Therapeutics Launch Check Ammonia Campaign to Highlight Critical Importance of Prompt, Accurate Blood Ammonia Testing for Diagnosing Urea Cycle Disorders
The National Urea Cycle Disorders Foundation (NUCDF) and Zevra Therapeutics (NasdaqGS: ZVRA) have launched the Check Ammonia Campaign in October to improve the diagnosis of urea cycle disorders (UCDs). The campaign aims to highlight the critical importance of prompt and accurate blood ammonia testing, which is essential for detecting elevated ammonia levels (hyperammonemia), a classic sign of UCDs.
The campaign website, checkammonia.com, offers resources on recognizing and testing for hyperammonemia. Quick diagnosis is important as toxic ammonia levels can rise rapidly in affected individuals, potentially leading to coma and death if left untreated. Newborns with severe UCDs typically become critically ill within 36-48 hours after birth, and it's estimated that up to 20% of sudden infant death syndrome (SIDS) cases may be due to undiagnosed inborn errors of metabolism like UCDs.
La National Urea Cycle Disorders Foundation (NUCDF) e Zevra Therapeutics (NasdaqGS: ZVRA) hanno lanciato la Campagna Check Ammonia in ottobre per migliorare la diagnosi dei disturbi del ciclo dell'urea (UCD). La campagna mira a sottolineare l'importanza critica di un tempestivo e accurato test del sangue per l'ammoniaca, essenziale per rilevare livelli elevati di ammoniaca (iperammoniemia), un segno classico degli UCD.
Il sito web della campagna, checkammonia.com, offre risorse su come riconoscere e testare l'iperammoniemia. Una diagnosi rapida è importante poiché i livelli tossici di ammoniaca possono aumentare rapidamente negli individui colpiti, portando potenzialmente a coma e morte se non trattati. I neonati con gravi UCD di solito diventano critici entro 36-48 ore dalla nascita, e si stima che fino al 20% dei casi di sindrome della morte improvvisa infantile (SIDS) possa essere dovuto a errori metabolici congeniti non diagnosticati come gli UCD.
La Fundación Nacional de Trastornos del Ciclo de Urea (NUCDF) y Zevra Therapeutics (NasdaqGS: ZVRA) lanzaron la Campaña Check Ammonia en octubre para mejorar el diagnóstico de los trastornos del ciclo de urea (UCD). La campaña busca resaltar la importancia crítica de una prueba rápida y precisa de la sangre para el amoníaco, que es esencial para detectar niveles elevados de amoníaco (hiperamonemia), un signo clásico de los UCD.
El sitio web de la campaña, checkammonia.com, ofrece recursos sobre cómo reconocer y probar la hiperamonemia. Un diagnóstico rápido es importante ya que los niveles tóxicos de amoníaco pueden aumentar rápidamente en los individuos afectados, lo que podría llevar a coma y muerte si no se trata. Los recién nacidos con UCD graves suelen enfermarse críticamente dentro de las 36-48 horas posteriores al nacimiento, y se estima que hasta el 20% de los casos de síndrome de muerte súbita del lactante (SIDS) pueden ser atribuidos a errores innatos del metabolismo no diagnosticados como los UCD.
국립 요소 순환 장애 재단(NUCDF)과 Zevra Therapeutics(NasdaqGS: ZVRA)는 요소 순환 장애(UCD)의 진단을 개선하기 위해 10월에 아모니아 점검 캠페인을 시작했습니다. 이 캠페인은 고수준의 혈액 아모니아 검사의 신속하고 정확한 중요성을 강조하는 것을 목표로 하며, 이는 UCD의 전형적인 징후인 높은 아모니아 수치(고암모니아혈증)를 발견하는 데 필수적입니다.
캠페인 웹사이트인 checkammonia.com은 고아모니아혈증을 인식하고 검사하는 방법에 대한 자료를 제공합니다. 독성 아모니아 수치는 영향을 받는 개인에서 빠르게 상승할 수 있으므로 신속한 진단이 중요하며, 치료하지 않으면 혼수 및 사망으로 이어질 수 있습니다. 심각한 UCD를 가진 신생아는 보통 출생 후 36-48시간 이내에 심각하게 아프며, SIDS(영아 돌연사 증후군) 사례 중 최대 20%가 UCD와 같은 진단되지 않은 선천성 대사 오류로 인해 발생할 수 있습니다.
La National Urea Cycle Disorders Foundation (NUCDF) et Zevra Therapeutics (NasdaqGS: ZVRA) ont lancé en octobre la Campagne Check Ammonia afin d'améliorer le diagnostic des troubles du cycle de l'urée (UCD). Cette campagne vise à souligner l'importance cruciale d'un test sanguin de l'ammoniaque, rapide et précis, qui est essentiel pour détecter des niveaux élevés d'ammoniaque (hyperammoniémie), un signe classique des UCD.
Le site web de la campagne, checkammonia.com, propose des ressources sur la reconnaissance et le test de l'hyperammoniémie. Un diagnostic rapide est important car les niveaux d'ammoniaque toxiques peuvent augmenter rapidement chez les individus touchés, pouvant mener à un coma et à la mort s'ils ne sont pas traités. Les nouveau-nés atteints de graves UCD deviennent souvent gravement malades dans les 36 à 48 heures suivant leur naissance, et il est estimé que jusqu'à 20 % des cas de syndrome de mort subite du nourrisson (SMSN) pourraient être dus à des erreurs métaboliques congénitales non diagnostiquées telles que les UCD.
Die National Urea Cycle Disorders Foundation (NUCDF) und Zevra Therapeutics (NasdaqGS: ZVRA) haben im Oktober die Check Ammonia Kampagne gestartet, um die Diagnose von Harnstoffzyklus-Störungen (UCD) zu verbessern. Die Kampagne zielt darauf ab, die entscheidende Bedeutung einer zeitnahen und genauen Blutammoniak-Testung hervorzuheben, die zur Feststellung erhöhter Ammoniakwerte (Hyperammonämie) wichtig ist, ein klassisches Zeichen für UCD.
Die Kampagnen-Website checkammonia.com bietet Ressourcen zur Erkennung und Testung von Hyperammonämie. Eine schnelle Diagnose ist wichtig, da toxische Ammoniakwerte bei betroffenen Personen schnell ansteigen können, was unbehandelt zu Koma und Tod führen kann. Neugeborene mit schweren UCDs werden in der Regel innerhalb von 36-48 Stunden nach der Geburt kritisch krank, und es wird geschätzt, dass bis zu 20% der Fälle von plötzlichem Kindstod (SIDS) auf undiagnostizierte angeborene Stoffwechselstörungen wie UCD zurückzuführen sein könnten.
- Zevra Therapeutics is partnering with NUCDF to raise awareness about urea cycle disorders
- The campaign aims to improve diagnosis and potentially save lives through proper blood ammonia testing
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Insights
The launch of the Check Ammonia Campaign by NUCDF and Zevra Therapeutics is a significant step towards improving the diagnosis of urea cycle disorders (UCDs). While this initiative is commendable from a healthcare perspective, its direct impact on Zevra Therapeutics' stock (ZVRA) is likely to be in the short term.
The campaign primarily focuses on raising awareness and improving diagnostic practices, which doesn't immediately translate to financial gains. However, it does position Zevra as a key player in the UCD space, potentially enhancing its reputation and market presence. This could indirectly benefit the company in the long run by:
- Increasing diagnosis rates, potentially expanding the market for UCD treatments
- Strengthening relationships with healthcare providers and patient advocacy groups
- Laying groundwork for future product launches or clinical trials related to UCD treatments
Investors should view this as a strategic move that may contribute to Zevra's long-term growth potential in the rare disease market, rather than expecting immediate financial impact. The campaign's success in improving UCD diagnosis could eventually lead to increased demand for related therapies, benefiting companies like Zevra that are invested in this space.
Patients, providers, and public are invited to help save lives by sharing messages and materials
PASADENA, Calif. and CELEBRATION, Fla., Oct. 09, 2024 (GLOBE NEWSWIRE) -- 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
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
FAQ
What is the Check Ammonia Campaign launched by NUCDF and Zevra Therapeutics (ZVRA)?
Why is blood ammonia testing important for diagnosing urea cycle disorders according to Zevra Therapeutics (ZVRA)?
What are the potential consequences of undiagnosed urea cycle disorders mentioned in Zevra Therapeutics' (ZVRA) campaign?
Where can people find resources about the Check Ammonia Campaign supported by Zevra Therapeutics (ZVRA)?
