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BridgeBio Launches MyAchonJourney, a New Resource for Families Navigating Achondroplasia

BridgeBio Launches MyAchonJourney, a New Resource for Families Navigating Achondroplasia

BridgeBio推出MyAchonJourney,這是一個新資源,爲家庭導航Achondroplasia提供幫助
BridgeBio Pharma ·  08/19 00:00

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.

2024年8月19日,加利福尼亞州帕洛阿爾託(GLOBE NEWSWIRE)—— BridgeBio Pharma,Inc.(納斯達克:BBIO)(BridgeBio)是一家專注於遺傳性疾病的商業階段生物製藥公司,QED Therapeutics是BridgeBio子公司,專注於開發治療骨發育不良的選擇。今天宣佈啓動了新亞可脫髮旅程的初始階段,這是一個新的在線資源,支持與骨性發育異常症狀相關的個人和家庭。

"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."

「MyAchonJourney幫助彌合醫療保健與日常生活體驗之間的差距,」尼莫爾斯兒童健康診所骨科手術部榮譽主席威廉·麥肯齊 (William Mackenzie)博士表示:「這個全面的資源的目標不僅是分享有關患有軟骨形成不全的醫療影響的知識,還爲家庭提供他們需要的工具,使他們能夠達到自主和保證,讓他們的孩子能夠茁壯成長,生活得充實和快樂。」

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.

MyAchonJourney的推出標誌着一個多階段計劃的開始,旨在爲軟骨形成不全症患者的家庭在他們的生活的每個階段提供持續,不斷髮展的支持。MyAchonJourney與骨骼發育不良社群的成員和領袖合作開發,旨在滿足爲患有軟骨形成不全的家庭提供教育和意識的重大需求。

"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."

「作爲患有軟骨形成不全的個人,我們的旅程非常廣泛和豐富多彩。我們社群的獨特性爲世界和社會提供了寶貴的視角,如果不堅持分享生活體驗的多樣性,這個寶貴的社群就會失去。像MyAchonJourney這樣的倡議對於提高意識、爲我們的社群提供知識和保證我們的聲音繼續被重視、尊重和認可是有幫助的,」美國小個子協會(Little People of America)生物技術委員會成員柯琳·吉奧弗雷達(Colleen Gioffreda)表示。

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:

在其初始階段,MyAchonJourney提供有關穿越孩子生命早期五年內醫療話題、心理社會問題和適應生活質量的指導,其中包括:

  • 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.
  • 懷孕和分娩:有關在出生前接受診斷,爲寶寶的到來做準備以及在家中的最初幾天應該注意什麼的信息。
  • 嬰幼兒期(出生-2歲):有關潛在的醫療問題、早期發育里程碑、家庭和服裝修改以及爲父母提供的基本支持和建議的信息。
  • 幼兒時期(3-5歲):有關識別支持行動不便的支持、家庭、學校和活動的適應以及心理社會資源的信息。

Subsequent phases to be shared on the website will encompass support and resources for older children, teenagers, and young adults.

網站上分享的後續階段將涵蓋爲年齡較大的兒童、青少年和年輕成年人提供支持和資源。

"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."

QED(BridgeBio的附屬機構)和BridgeBio致力於推進教育和認識,超越軟骨形成不全症患者的治療決策,”QED全球患者倡導高級主任安妮·李·格魯梅特(Anne Lee Grumet)表示:「我們很感激社區的協作努力,開發了一個全面的資源,幫助患有軟骨形成不全症的個人和家庭從出生到成年期全面了解他們所需要的信息。我們的MyAchonJourney計劃是提供一個可靠和包容的平台,爲人們及其所愛的人提供實用信息,使他們能夠自信地過上充實的生活。」

A special thank you to the dedicated task force members whose expertise and commitment were pivotal in developing MyAchonJourney:

特別感謝專門的工作小組成員,他們的專業知識和承諾在發展MyAchonJourny計劃時發揮了關鍵作用:

  • 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
  • 錢德勒·克魯斯(Chandler Crews),The Chandler Project的創始人
  • 柯琳·吉奧弗雷達,約翰霍普金斯大學臨床運營計劃管理員和美國小個子協會生物技術委員會成員
  • 黛博拉·克拉考,UCLA大衛傑弗遜醫學院人類遺傳學、兒科和骨科手術主任及教授
  • 金柏利·芬,佛羅里達南區浸會健康公司的職業治療師
  • 克里斯汀·德安德拉德(Kristen DeAndrade),小腿大心臟基金會的創始人
  • 馬裏·格洛夫斯(Mari Groves),約翰霍普金斯醫院兒科神經外科分部副教授
  • 米歇爾·克勞斯(Michelle Kraus),紐約公益律師協會的高級社會工作者
  • 穆尼拉·沙迷姆(Munira Shamim),Growing Stronger的聯合創始人和家長
  • 尼莫瓦兒童健康診所骨科手術系榮譽主席威廉·麥肯齊(William Mackenzie)表示:「MyAchonJourney是一項意味深長的、性價比高的和對個人和家庭產生深遠影響的計劃。隨着我們對軟骨形成不全症的理解不斷增長,這樣的倡議將爲改善生活質量、鞏固社群並進一步推進醫學和科學的發展奠定基礎。」

For more information and to explore the new website, visit MyAchonJourney.

訪問MyAchonJourney網站以了解更多信息。

About BridgeBio Pharma, Inc.

關於BridgeBio Pharma股份有限公司。

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 (BridgeBio)是一家商業化生物製藥公司,致力於發現、創建、測試和提供轉化性藥物以治療遺傳性疾病患者。BridgeBio的開發項目管道從早期科學到先進的臨床試驗。BridgeBio成立於2015年,其由經驗豐富的藥物發現家、開發人員和創新者組成的團隊致力於將遺傳醫學的進步儘快應用於幫助患者。欲了解更多信息,請訪問bridgebio.com並關注我們的LinkedIn、Twitter和Facebook頁面。

BridgeBio Pharma, Inc. Forward-Looking Statements

BridgeBio Pharma, Inc.前瞻性聲明

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.

本新聞稿包含前瞻性陳述。本新聞稿中的陳述可能包括不是歷史事實的陳述,並被認爲是《1933年證券法》第27A條規定的前瞻性,以及《1934年證券交易法》第21E條修改的(《證券交易法》),其通常的特徵是使用諸如「預計」、「相信」、「繼續」、「估計」、「期望」、「希望」、「打算」、「可能」、「計劃」、「項目」、「仍然」、「尋求」、「應該」、「將」和此類詞語或類似表達方式。我們希望這些前瞻性陳述將受到《證券法》第27A條和《證券交易法》第21E條規定的前瞻性陳述的安全港規定的保護。這些前瞻性陳述基於我們目前掌握的信息以及我們作出的假設。雖然公司相信其計劃、意圖、期望和策略如反映或由這些前瞻性陳述所提示的那樣是合理的,但我們無法保證這些計劃、意圖、期望或策略將得到實現或實現。此外,實際結果可能會與前瞻性陳述中所描述的結果存在實質性差異,並受到許多風險、不確定性和假設的影響,包括但不限於正在進行和計劃的MyAchonJourney平台的設計和成功、公司合作的持續成功、公司獲得額外資金的能力,以及列於公司最近的《年度報告10-k》和公司在美國證券交易委員會的其他文件中的《風險因素》部分所規定的那些風險。此外,我們在一個非常競爭和快速變化的環境中運營,其中會不時出現新的風險。這些前瞻性陳述基於公司管理層在本新聞稿發表之日的當前預期和信念,並且受一定風險和不確定因素的影響,這些因素可能導致實際結果與前瞻性陳述中描述的結果存在實質性差異。除適用法律另有規定外,我們不承諾公開更新任何前瞻性陳述,無論是因爲新信息、未來事件還是其他因素。

BridgeBio Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220

BridgeBio聯繫人:
Vikram Bali
contact@bridgebio.com
(650)-789-8220

QED Contact:
Anne Lee Grumet
Sr. Director, Global Patient Advocacy
PatientAdvocacyQED@bridgebio.com

QED聯繫人:
Anne Lee Grumet
全球患者倡導高級董事
PatientAdvocacyQED@bridgebio.com


声明:本內容僅用作提供資訊及教育之目的,不構成對任何特定投資或投資策略的推薦或認可。 更多信息
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