Press release
Zevra Therapeutics to Participate in the 2023 NNPDF Family Support and Medical Conference
CELEBRATION, Fla., July 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"CELEBRATION, Fla., July 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, today announced their support of the upcoming National Niemann-Pick Disease Foundation’s (NNPDF) Family Support and Medical Conference. Members of the Zevra team will attend this event which is being held in Orlando, Florida, from July 20 through July 22, 2023. In addition to providing a corporate update, Zevra plans to lead a family working group session to gain insights from caregivers on important topics facing the rare disease community. Two research posters will also be presented during the event, with a focus on previously published information regarding the validity of the 5-Domain NPC Clinical Severity Scale (5D NPCCSS) as a clinical outcomes assessment measure, and the correlation between the 5D NPCCSS and corresponding items of the Scale for the Assessment and Rating of Ataxia (SARA). “NNPDF’s Family Support and Medical Conference is a significant event for the NPC community that brings together patients, caregivers, clinicians and researchers to promote much needed awareness, understanding and information about services and support available and education about potential treatments for this devastating disease,” said Christal Mickle, President, interim Chief Executive Officer and Chief Development Officer of Zevra. “The Zevra team is proud to sponsor the NNPDF and their work supporting the patients and their families who continue to inspire us with their strength and hope. We look forward to being a part of this year's event.” About Niemann-Pick disease type C (NPC): Niemann-Pick disease type C (NPC) is an ultra-rare and progressive, neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes which are responsible for making lysosomal proteins and is an autosomal recessive trait. Both children and adults can be affected by NPC with varying age of onset, rate of progression and presents differently in each person. Those living with NPC lose independence due to physical and cognitive limitations, with k...