BridgeBio Launches MyAchonJourney, a New Resource for Families Navigating Achondroplasia

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 , 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 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, 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, 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 :

• 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 .

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  and follow us on ,  and .

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



(650)-789-8220

QED Contact:

Anne Lee Grumet

Sr. Director, Global Patient Advocacy