Osteogenesis imperfecta

Ultragenyx to Present Setrusumab (UX143) Update at ASBMR 2023 Including New Data from Phase 2/3 Orbit Study in Osteogenesis Imperfecta (OI)

Retrieved on: 
Monday, October 9, 2023
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The company will also present information on the Phase 3 Cosmic study design comparing setrusumab to intravenous bisphosphonate (IV-BP) in patients aged 2 to

Key Points: 
  • The company will also present information on the Phase 3 Cosmic study design comparing setrusumab to intravenous bisphosphonate (IV-BP) in patients aged 2 to
  • Details of the setrusumab presentations are as follows:
    Abstract Title: Evaluating Setrusumab for the Treatment of Osteogenesis Imperfecta: Phase 2 Data from the Phase 2/3 ORBIT Study (Lewiecki, E.M. et al.)
  • Abstract Title: Cosmic: An open-label, randomized, active-controlled, phase 3 study of setrusumab compared with bisphosphonates in pediatric subjects with osteogenesis imperfecta (Krolczyk, S. et al.)
  • The companies previously announced positive data from the dose-selection Phase 2 portion of the Orbit study showing that setrusumab rapidly induced bone production in OI-affected patients.

Ultragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023

Retrieved on: 
Thursday, October 5, 2023
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NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m.

Key Points: 
  • NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m.
  • ET on Monday, October 16, 2023.
  • Ultragenyx leaders and external experts will provide an update on the company’s development pipeline.
  • An archived version of the remarks will also be available through the Ultragenyx website.

The Dream Machine: When Fiction Exceeds Reality

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Wednesday, September 27, 2023
Dream Machine, Convalescence, Anxiety, Hospital, Osteogenesis imperfecta, Leg, Shriners Hospital for Children – Canada, Language, Pain, School, Nursing Research, Patient, Shriners Hospitals for Children, RN, Knowledge, Rare, Health, Mental health, OI, Entertainment, Nursing, Child, Shriners

The Dream Machine, written by Candace Amarante and wonderfully illustrated by Dave Reed, comes from the Nursing Research Department of Shriners Hospitals for Children - Canada.

Key Points: 
  • The Dream Machine, written by Candace Amarante and wonderfully illustrated by Dave Reed, comes from the Nursing Research Department of Shriners Hospitals for Children - Canada.
  • She therefore decides to share her secret with her little sister: The Dream Machine.
  • Like Ashling, I used my dream machine to draw on reality and fantasy to bring this story to life.
  • When children ask me what my favourite book is, I now answer, The Dream Machine,” says author Candace Amarante.

The Dream Machine: When Fiction Exceeds Reality

Retrieved on: 
Wednesday, September 27, 2023
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MONTREAL, Sept. 27, 2023 (GLOBE NEWSWIRE) -- Attend the launch of The Dream Machine, a book by Candace Amarante, beautifully illustrated by Dave Reed!

Key Points: 
  • MONTREAL, Sept. 27, 2023 (GLOBE NEWSWIRE) -- Attend the launch of The Dream Machine, a book by Candace Amarante, beautifully illustrated by Dave Reed!
  • The Shriners Hospital for Children - Canada, in the company of Maria Farella, whose philanthropic successes and commitment to the mental health of young people are well known (e.g.
  • President of the fundraising event Notte in Bianco since 2008), will be celebrating the release of this excellent tool from its Nursing Research Department at a press conference.
  • Author Candace Amarante, Illustrator Dave Reed, Principal Investigator Argerie Tsimicalis, Ph.D., Drs.

Mereo BioPharma Reports Interim Financial Results for the Six Months Ended June 30, 2023 and Provides Corporate Update

Retrieved on: 
Thursday, September 7, 2023
Ageing, Osteogenesis imperfecta, Patient, OI, Sale, Pharmaceutical industry

LONDON, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its unaudited interim financial results for the six months ended June 30, 2023 and provided an update on recent corporate highlights.

Key Points: 
  • LONDON, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its unaudited interim financial results for the six months ended June 30, 2023 and provided an update on recent corporate highlights.
  • All 24 patients are now enrolled in a Phase 2 open-label extension study, with additional data expected to be shared by Ultragenyx in mid-October 2023.
  • Administrative expenses increased by £0.7 million, or 8%, from £8.8 million for the six months ended June 30, 2022 to £9.5 million for the six months ended June 30, 2023.
  • Total ADS equivalents at August 31, 2023 were 140,176,617, with each ADS representing five ordinary shares of the Company.

Phoenix Children's Expands Pediatric Orthopedic Surgery Team with Hire of Globally Renowned Brittle Bone Expert

Retrieved on: 
Monday, September 11, 2023
American Society for Bone and Mineral Research, Sports medicine, Missouri State University, University, Nationwide Children's Hospital, Body composition, University of Nebraska Medical Center, Phoenix Children's Hospital, Elective surgery, University of Nebraska–Lincoln, Quality of life, Hope, Nebraska Medicine, Osteoblast, Osteogenesis imperfecta, Collagen, type I, alpha 1, MBA, Research, Chronic kidney disease-mineral and bone disorder, National Association Medical Staff Services, Business administration, Jansen, Diving Medical Advisory Council, MD, Patient, Lincoln, Nebraska, Medical Center, Hospital, AI, Saint Louis University, Society, Dentistry, Nursing, Medical device, Orthopedic surgery, Child

Maegen Wallace, MD, MBA , a pediatric orthopedic surgeon and expert in osteogenesis imperfecta, or brittle bone disease, joined the Herbert J. Louis Center for Orthopedics and Sports Medicine at Phoenix Children's last month.

Key Points: 
  • Maegen Wallace, MD, MBA , a pediatric orthopedic surgeon and expert in osteogenesis imperfecta, or brittle bone disease, joined the Herbert J. Louis Center for Orthopedics and Sports Medicine at Phoenix Children's last month.
  • At the Herbert J. Louis Center for Orthopedics and Sports Medicine at Phoenix Children's, Dr. Wallace will focus on lower-extremity issues, osteogenesis imperfecta and other rare skeletal diseases .
  • On the research front, she will continue her work to define best clinical practices for patients with brittle bone disease and uncover new treatment options.
  • "Phoenix Children's emphasis on research and the chance to work once again with Dr. Halanski are what drew me to Phoenix Children's," said Dr. Wallace.

Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update

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Thursday, August 3, 2023
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NOVATO, Calif., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended June 30, 2023 and provided its financial guidance for the year.

Key Points: 
  • For the second quarter of 2023, Ultragenyx reported net loss of $159.8 million, or $2.25 per share basic and diluted, compared with a net loss for the second quarter of 2022 of $158.2 million, or $2.26 per share, basic and diluted.
  • Howard Horn was appointed as Ultragenyx’s Chief Financial Officer (CFO) and Executive Vice President, Corporate Strategy, effective October 16, 2023.
  • Ultragenyx will host a conference call today, Thursday, August 3, 2023, at 2 p.m. PT/5 p.m.
  • ET to discuss the second quarter 2023 financial results and provide a corporate update.

Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, May 4, 2023
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NOVATO, Calif., May 04, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023.

Key Points: 
  • This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
  • In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143.
  • No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023.
  • ET to discuss the first quarter 2023 financial results and provide a corporate update.

OrthoPediatrics Corp. Leading Sponsorship at Annual EPOS Meeting

Retrieved on: 
Friday, March 31, 2023
Osteogenesis imperfecta, Elective surgery, Orthopediatrics, KIDS, EPOS, Dentistry

WARSAW, Ind., March 31, 2023 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS) a company focused exclusively on advancing the field of pediatric orthopedics, today announced its leading sponsorship at the European Pediatric Orthopaedic Society (“EPOS”) annual meeting in Krakow, Poland.

Key Points: 
  • WARSAW, Ind., March 31, 2023 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS) a company focused exclusively on advancing the field of pediatric orthopedics, today announced its leading sponsorship at the European Pediatric Orthopaedic Society (“EPOS”) annual meeting in Krakow, Poland.
  • The company hosted an exhibit for OrthoPediatrics, Pega Medical, and MD Orthopaedics as well as a surgeon luncheon.
  • OrthoPediatrics’ President & CEO, David Bailey, commented, “We are excited to be in Poland with our surgeon customers from all over Europe.
  • The presentation highlighted case studies and engaged EPOS attendees in discussion of their experiences and best practices.

Mereo BioPharma Reports Full Year 2022 Financial Results and Recent Highlights

Retrieved on: 
Tuesday, March 28, 2023
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LONDON, March 28, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the year ended December 31, 2022 and provided an update on recent corporate highlights.

Key Points: 
  • LONDON, March 28, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its financial results for the year ended December 31, 2022 and provided an update on recent corporate highlights.
  • “In 2022, we made important progress in the ongoing advancement of our core rare disease programs.
  • Full year 2022 research and development expenses were £25.0 million, compared to £23.6 million, in 2021, an increase of £1.4 million, or 6%.
  • Total ADSs outstanding at December 31, 2022 were approximately 119 million, with each ADS representing five ordinary shares of the Company.