Educational Episode to Highlight Symptoms of ENPP1 Deficiency and the Importance of Early Diagnosis
BOSTON, March 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today that ENPP1 deficiency will be featured on The Balancing Act® as part of the show’s recurring “Behind the Mystery of Rare and Genetic Diseases” series to raise awareness for this rare, debilitating, and life-threatening mineralization disease for which there are currently no approved treatment options.
The episode will feature Janine Hicks, a patient living with ENPP1 deficiency; her parents, Donna and Peter Hicks; and Professor Zulf Mughal, a treating physician and researcher and Consultant in Pediatric Bone Disorders, Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust. The segment will cover scientific and medical aspects of ENPP1 deficiency, the importance of making an accurate diagnosis, and the daily challenges faced by patients and families living with this debilitating disease.
“Many rare diseases like ENPP1 deficiency are not easily recognized or well-understood and patients face long delays in getting an accurate diagnosis. It is our hope that this segment will increase awareness of this devastating disease so that patients can receive an accurate diagnosis and appropriate medical treatment as we work to develop a therapy to treat patients who currently lack options,” said Axel Bolte, co-founder, president, and chief executive officer of Inozyme. “We are extremely grateful to Janine, Donna, Peter, and Dr. Mughal for telling this powerful story and bringing a voice to the many challenges that patients and families dealing with ENPP1 deficiency face every day.”
“At GACI Global, we provide information about the complications that can occur with ENPP1 and ABCC6 deficiency and seek to bring hope to families impacted by these rare and life-threatening conditions,” said Christine O’Brien and Liz Molloy, co-presidents of GACI Global. “Our mission is to raise awareness of these rare conditions through research and education, so we welcomed this opportunity to work with Inozyme on this initiative. We, too, are grateful for everyone’s participation in the development of this segment.”
For the past 10 years, Behind the Mystery has spotlighted undiagnosed, misdiagnosed, and complex medical disorders by partnering with pharmaceutical and biotechnology companies who are on a mission to educate the public to bring earlier diagnosis and treatment and provide a sense of community for people living with these disorders.
The segment will air on The Balancing Act® on Lifetime® on Monday, March 15, 2021 at 7:30 a.m., ET/PT and again on Wednesday, March 24, 2021 at 7:30 a.m. ET/PT, after which it will be shown in syndication.
About ENPP1 Deficiency
The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. Mutations in the ABCC6 gene can also cause an infantile onset of the disease called GACI Type 2. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.
About The Balancing Act
The Balancing Act® is a morning show created and produced by BrandStar that offers sensible solutions and essential information in a fun, entertaining format; providing resources to help people do life better. The Balancing Act features everything from delicious recipes, style makeovers and dream getaways to parenting tips and the latest news in health and wealth. Tune in to The Balancing Act weekdays at 7:30 a.m. (ET/PT) on Lifetime® and find all previously aired episodes on TheBalancingAct.com.
About GACI Global
GACI Global is a nonprofit patient advocacy group whose mission is to connect families affected by Generalized Arterial Calcification of Infancy of Hypophosphatemic Rickets caused by ENPP1 or ABCC6 deficiencies to each other and to the medical community. The organization strives to provide current educational resources and supports ongoing research. The goal of this 100% volunteer run organization is to provide not only information about what complications can occur due to ENPP1 and ABCC6 deficiencies, but to provide hope for families impacted by the condition around the world.
About Inozyme Pharma
Inozyme Pharma, Inc. (Nasdaq: INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
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Axel Bolte, co-founder, president, and chief executive officer
Alex Van Rees
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