Results 41 to 50 of about 534 (161)

O22: A randomized controlled trial of vosoritide in infants and toddlers with achondroplasia*

Genetics in Medicine Open, 2023
Carlos Bacino, Ravi Savarirayan, William Wilcox, Paul Harmatz, John Phillips, Lynda Polgreen, Louise Tofts, Keiichi Ozono, Paul Arundel, Melita Irving, Donald Basel, Michael Bober, Joel Charrow, Hiroshi Mochizuki, Yumiko Kotani, Howard Saal, George Jeha, Lynn Han, Elena Fisheleva, Alice Huntsman-Labed, Jonathan Day   +20 more
doaj   +2 more sources

SAT-LB18 A Randomized Controlled Trial of Vosoritide in Children With Achondroplasia [PDF]

, 2020
Background: Achondroplasia is a disorder caused by specific mutations in the gene encoding the fibroblast growth factor receptor 3 (FGFR3) protein. Open-label, phase 2 trials in children with achondroplasia showed that administration of vosoritide, an ...
Tofts, Louise, Savarirayan, Ravi, Kagami, Shoji, Ginebreda, Ignacio, Ozono, Keiichi, Mohnike, Klaus, Huntsman-Labed, Alice, Polgreen, Lynda E, Charrow, Joel, Irving, Melita, Wilcox, William, Hoernschmeyer, Daniel, Luna-Gonzáles, Felipe, Bober, Michael B, Jayaram, Kala, Saal, Howard M, Arundel, Paul, White, Klane, Porco, Dania M, Harmatz, Paul, Leiva-Gea, Antonio, Fisheleva, Elena, Rutsch, Frank, Yasui, Natsuo, Basel, Donald, Hoover-Fong, Julie, Alanay, Yasemin, Bacino, Carlos A, Mochizuki, Hiroshi, Day, Jonathan, Font, Rosendo Ullot   +30 more
core   +1 more source

Vosoritide therapy in children with achondroplasia under 5 years of age. [PDF]

Transl Pediatr
Kitoh H.
europepmc   +3 more sources

Assessment of the efficacy of vosoritide therapy in children with achondroplasia in clinical trials. [PDF]

Transl Pediatr
Wrobel W, Ben-Skowronek I.
europepmc   +3 more sources

FRD-2023-0002: A caregiver and physician perspective on the role of vosoritide in the treatment of achondroplasia: an interview with Mary Andrews and Melita Irving.mp3

, 2023
A caregiver and physician perspective on the role of vosoritide in the treatment of achondroplasia: an interview with Mary Andrews and Melita ...
Mary Andrews (3245388), Melita Irving (198804)   +1 more
core   +1 more source

Orthodontic and Maxillofacial Surgery Treatment in Achondroplasia for Orofacial Alterations: A Systematic Review and Preliminary Age‐Stratified Guidelines

Orthodontics &Craniofacial Research, Volume 29, Issue 4, Page 593-610, August 2026.
ABSTRACT This systematic review aimed to collect and appraise the clinical outcomes of all orthopaedic, orthodontic and surgical interventions in ACH patients. Following PROSPERO protocol, multiple database sources were searched to December 2024 with no language restrictions for (i) genetically confirmed ACH; (ii) any orthodontic/orthopaedic ...
Marco Farronato, Maria Francesca Bedeschi, Cristina Grippaudo, Gianluca Martino Tartaglia, Berardo Rinaldi, Cinzia Maspero   +5 more
wiley   +1 more source

Homozygous Achondroplasia With Long‐Term Survival: Growth Patterns, Medical Interventions, and Practice Implications

American Journal of Medical Genetics Part A, Volume 200, Issue 6, Page 1372-1377, June 2026.
ABSTRACT Homozygous achondroplasia is widely considered perinatal lethal by the medical community. In this case series, we report two children from a single family with longer‐term survival. One child lived for 17 months and the other was 60 months at the time of publication.
Hannah Singerline, Jason Laufman, Kimberly Wallis, Michelle Merrill   +3 more
wiley   +1 more source

Analysis of Real‐World Data Utilization in the Orphan Drug Approval Process: Focusing on New Drug Marketing Applications Submitted to the FDA

Clinical Pharmacology &Therapeutics, Volume 119, Issue 5, Page 1340-1349, May 2026.
In the field of rare diseases—where traditional clinical trials are often impractical—real‐world data (RWD) have emerged as a scientifically valid alternative to support regulatory decision making. This study systematically evaluates the utilization of RWD in orphan drug approvals by the FDA Center for Drug Evaluation and Research (CDER) over the past ...
Minji Kim, Eunjin Hong
wiley   +1 more source

Evaluating the impact of vosoritide on complications of achondroplasia

Genetics in Medicine
Impaired endochondral bone growth in people with achondroplasia causes complications that impair function, cause pain, require surgery, and contribute to mortality. Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH ...
Ravi Savarirayan, Jacqueline T. Hecht, Kala Jayaram, Sheetal Ingole, Anne Dee, Julie Hoover-Fong   +5 more
openaire   +2 more sources

A Comparison of Regional Decisions for Doses and Administrations of New Drugs: Concordance, Discordance, and Dependencies

Clinical Pharmacology &Therapeutics, Volume 119, Issue 5, Page 1350-1361, May 2026.
Regional differences in approved drug dosages and administration are shaped by complex factors, including clinical data, prior regulatory decisions, and region‐specific considerations. This study investigated how such factors are associated with variations in approved doses across the United States Food and Drug Administration (FDA), European Medicines
Sachiko Mita, Shunsuke Ono
wiley   +1 more source