BridgeBio Launches MyAchonJourney, a New Resource for Families Navigating Achondroplasia
August 19 2024 - 6:30AM
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
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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
Balicontact@bridgebio.com(650)-789-8220
QED Contact: Anne Lee GrumetSr. Director,
Global Patient AdvocacyPatientAdvocacyQED@bridgebio.com
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