BridgeBio Launches MyAchonJourney, a New Resource for Families Navigating Achondroplasia
BridgeBio Pharma (Nasdaq: BBIO) and its affiliate QED Therapeutics have launched MyAchonJourney, an online resource for families navigating achondroplasia. Developed in collaboration with skeletal dysplasia community leaders, the initiative aims to provide education and support for families affected by achondroplasia.
The initial phase of MyAchonJourney offers guidance on medical topics, psychosocial issues, and quality-of-life adaptations for children up to five years old. It covers pregnancy, birth, infancy, toddlerhood, and early childhood, with plans to expand resources for older children, teenagers, and young adults in future phases.
The platform was created with input from a task force of experts, including medical professionals and community advocates, to ensure comprehensive and reliable information for families navigating life with achondroplasia.
BridgeBio Pharma (Nasdaq: BBIO) e la sua affiliata QED Therapeutics hanno lanciato MyAchonJourney, una risorsa online per le famiglie che affrontano l'achondroplasia. Sviluppata in collaborazione con i leader della comunità delle displasie scheletriche, l'iniziativa mira a fornire istruzione e supporto alle famiglie colpite dall'achondroplasia.
La fase iniziale di MyAchonJourney offre indicazioni su argomenti medici, questioni psicosociali e adattamenti per la qualità della vita per bambini fino a cinque anni. Copre la gravidanza, la nascita, l'infanzia, la prima infanzia e la prima infanzia, con piani per espandere le risorse per bambini più grandi, adolescenti e giovani adulti nelle fasi future.
La piattaforma è stata creata con il contributo di un gruppo di esperti, tra cui professionisti medici e sostenitori della comunità, per garantire informazioni complete e affidabili per le famiglie che affrontano la vita con l'achondroplasia.
BridgeBio Pharma (Nasdaq: BBIO) y su filial QED Therapeutics han lanzado MyAchonJourney, un recurso en línea para familias que navegan la acondroplasia. Desarrollada en colaboración con líderes de la comunidad de displasias esqueléticas, la iniciativa tiene como objetivo proporcionar educación y apoyo a las familias afectadas por la acondroplasia.
La fase inicial de MyAchonJourney ofrece orientación sobre temas médicos, problemas psicosociales y adaptaciones en la calidad de vida para niños de hasta cinco años. Cubre el embarazo, el nacimiento, la infancia, la niñez temprana y la primera infancia, con planes para expandir los recursos para niños mayores, adolescentes y jóvenes adultos en fases futuras.
La plataforma fue creada con la contribución de un grupo de expertos, incluidos profesionales médicos y defensores de la comunidad, para garantizar información completa y confiable para las familias que navegan la vida con acondroplasia.
BridgeBio Pharma (Nasdaq: BBIO)와 그 자회사 QED Therapeutics는 MyAchonJourney를 출시했습니다. 이는 짧은 고관절증을 겪고 있는 가족들을 위한 온라인 자원입니다. 골격 이형성증 커뮤니티 리더들과 협력하여 개발된 이 이니셔티브의 목표는 교육 및 지원을 제공하는 것입니다.
MyAchonJourney의 초기 단계는 의료 주제, 심리사회적 문제 및 생활의 질을 위한 조정에 대한 가이드를 제공합니다. 이 가이드는 임신, 출산, 유아 및 조기 아동기를 포함하며, 향후 단계에서 나이가 많은 아이들, 청소년 및 젊은 성인들을 위한 자료를 확장할 계획입니다.
이 플랫폼은 의료 전문가 및 커뮤니티 옹호자들로 구성된 전문가 태스크 포스의 의견을 반영하여 만들어져, 짧은 고관절증과 함께하는 삶을 탐색하는 가족들을 위한 포괄적이고 신뢰할 수 있는 정보를 보장합니다.
BridgeBio Pharma (Nasdaq: BBIO) et sa filiale QED Therapeutics ont lancé MyAchonJourney, une ressource en ligne pour les familles confrontées à l'achondroplasie. Développée en collaboration avec les leaders de la communauté des dysplasies squelettiques, l'initiative vise à fournir éducation et soutien aux familles touchées par l'achondroplasie.
La phase initiale de MyAchonJourney propose des conseils sur des sujets médicaux, des problèmes psychosociaux et des adaptations de qualité de vie pour les enfants jusqu'à cinq ans. Elle couvre la grossesse, la naissance, la petite enfance et la petite enfance, avec des projets d'expansion des ressources pour les enfants plus âgés, les adolescents et les jeunes adultes dans les phases futures.
La plateforme a été créée avec l'apport d'un groupe d'experts, comprenant des professionnels de la santé et des défenseurs de la communauté, afin de garantir des informations complètes et fiables pour les familles vivant avec l'achondroplasie.
BridgeBio Pharma (Nasdaq: BBIO) und seine Tochtergesellschaft QED Therapeutics haben MyAchonJourney gestartet, eine Online-Ressource für Familien, die sich mit Achondroplasie auseinandersetzen. Die Initiative wurde in Zusammenarbeit mit Fachleuten aus der Gemeinschaft der Skelettdysplasien entwickelt und zielt darauf ab, Bildung und Unterstützung für betroffene Familien bereitzustellen.
Die erste Phase von MyAchonJourney bietet Orientierung zu medizinischen Themen, psychosozialen Fragen und Anpassungen der Lebensqualität für Kinder bis fünf Jahre. Sie behandelt Schwangerschaft, Geburt, Säuglingsalter, Kleinkindalter und frühe Kindheit und plant, die Ressourcen in zukünftigen Phasen für ältere Kinder, Jugendliche und junge Erwachsene zu erweitern.
Die Plattform wurde mit der Mitwirkung einer Expertengruppe, die aus medizinischen Fachkräften und Interessenvertretern der Gemeinschaft bestand, erstellt, um umfassende und verlässliche Informationen für Familien bereitzustellen, die das Leben mit Achondroplasie meistern.
- Launch of a new educational resource for families affected by achondroplasia
- Collaboration with skeletal dysplasia community leaders and experts in developing the platform
- Comprehensive coverage of various aspects of living with achondroplasia, from birth to early childhood
- None.
PALO ALTO, Calif., Aug. 19, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, and QED Therapeutics, the BridgeBio affiliate focused on developing treatment options for skeletal dysplasias, today announced the launch of the initial phase of MyAchonJourney, a new online resource to support individuals and families living with achondroplasia.
"MyAchonJourney helps bridge the gap between medical care and daily life experiences," says William Mackenzie, M.D., Emeritus Chairman of the Department of Orthopedic Surgery at Nemours Children’s Health. “The goal of this comprehensive resource is not only to share knowledge about the medical impacts of living with achondroplasia, but to provide families with the tools they need to empower independence and assurance that their children can thrive, living full and happy lives.”
The launch of MyAchonJourney marks the start of a multi-phase initiative designed to provide ongoing, evolving support tailored to the unique needs of families at every stage of their lives with achondroplasia. Developed in collaboration with members and leaders of the skeletal dysplasia community, MyAchonJourney intends to fulfill a significant need of providing education and awareness for families with achondroplasia.
"Our journey as individuals with achondroplasia is broad and colorful. The uniqueness of our community offers an invaluable perspective of the world and society, and would be lost if not for the commitment to share the benefits of diversity in lived experiences,” said Colleen Gioffreda, Little People of America Biotech Committee Member. “An initiative like MyAchonJourney is a helpful resource in raising awareness, empowering our community with knowledge, and ensuring that our voices continue to be valued, respected, and validated."
In its initial phase, MyAchonJourney provides guidance on navigating medical topics, psychosocial issues, and quality-of-life adaptations through the first five years of a child's life, including:
- Pregnancy and Birth: Information on receiving a diagnosis before birth, planning for the baby’s arrival, and what to expect in the initial days at home.
- Infancy and Toddlerhood (Birth-2 Years): Information on potential medical issues, early developmental milestones, home and clothing modifications, and essential advocacy tips for parents.
- Early Childhood (3-5 Years): Information on identifying support for mobility challenges; adaptations for home, school, and activities; and psychosocial resources.
Subsequent phases to be shared on the website will encompass support and resources for older children, teenagers, and young adults.
"QED and BridgeBio are committed to advancing education and awareness that goes beyond treatment decision-making for individuals living with achondroplasia," says Anne Lee Grumet, QED's Senior Director of Global Patient Advocacy. "We are grateful for the collaborative efforts of the community to develop a comprehensive resource that helps individuals and families navigate life with achondroplasia from birth through adulthood. Our intention for MyAchonJourney is to deliver a reliable and inclusive platform that equips people and their loved ones with practical information, empowering them to confidently lead enriching lives."
A special thank you to the dedicated task force members whose expertise and commitment were pivotal in developing MyAchonJourney:
- Chandler Crews, Founder, The Chandler Project
- Colleen Gioffreda, Clinical Operations Program Administrator, Johns Hopkins University and Little People of America Biotech Committee Member
- Deborah Krakow, M.D., Professor and Chair of the Department of Obstetrics and Gynecology; Professor of Human Genetics, Pediatrics and Orthopaedic Surgery; Department of Human Genetics, David Geffen School of Medicine at UCLA
- Kimberly Fine, M.S., OTR/L, Occupational Therapist, Baptist Health South Florida
- Kristen DeAndrade, Founder, Little Legs Big Heart Foundation
- Mari Groves, M.D., Associate Professor of Neurosurgery, Division of Pediatric Neurosurgery, Johns Hopkins Hospital
- Michelle Kraus, MSW, Senior Social Worker, New York Lawyers for the Public Interest
- Munira Shamim, J.D., Co-Founder and Parent, Growing Stronger
- William Mackenzie, M.D., Emeritus Chairman of the Department of Orthopedic Surgery, Nemours Children’s Health
For more information and to explore the new website, visit MyAchonJourney.
About BridgeBio Pharma, Inc.
BridgeBio Pharma (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015, and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook.
BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to fulfilling a significant need through the MyAchonJourney platform for knowledge and awareness for individuals and families living with achondroplasia, are based on the information currently available to us and on assumptions we have made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to the design and success of the ongoing and planned MyAchonJourney platform, the continuing success of the Company’s collaborations, the Company’s ability to obtain additional funding, as well as those risks set forth in the Risk Factors section of the Company’s most recent Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
BridgeBio Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220
QED Contact:
Anne Lee Grumet
Sr. Director, Global Patient Advocacy
PatientAdvocacyQED@bridgebio.com
FAQ
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