Results 61 to 70 of about 534 (161)

Dose Determinations at Drug Approval Reviews: FDA‐Approved Drugs in Past 5 Years

Clinical Pharmacology &Therapeutics, Volume 117, Issue 6, Page 1743-1753, June 2025.
Drug dose appropriateness is one of the most discussed issues in regulatory reviews. We analyzed dose determinations during Food and Drug Administration (FDA) drug reviews to determine whether there were changes between the proposed and approved doses of new molecular entities (NMEs), including cases where postmarketing dose‐finding studies were ...
Sachiko Mita, Shunsuke Ono
wiley   +1 more source

A registry of achondroplasia: a 6-year experience from the Czechia and Slovak Republic

Orphanet Journal of Rare Diseases, 2022
Background Achondroplasia (ACH) is one of the most prevalent genetic forms of short-limbed skeletal dysplasia, caused by gain-of-function mutations in the receptor tyrosine kinase FGFR3.
Martin Pesl, Hana Verescakova, Linda Skutkova, Jana Strenkova, Pavel Krejci   +4 more
doaj   +1 more source

Escherichia coli in the production of biopharmaceuticals

Biotechnology and Applied Biochemistry, Volume 72, Issue 2, Page 528-541, April 2025.
Abstract Escherichia coli has shouldered a massive workload with the discovery of recombinant DNA technology. A new era began in the biopharmaceutical industry with the production of insulin, the first recombinant protein, in E. coli and its use in treating diabetes. After insulin, many biopharmaceuticals produced from E. coli have been approved by the
İbrahim İncir, Özlem Kaplan
wiley   +1 more source

PMDA Perspective on Use of Real‐World Data and Real‐World Evidence as an External Control: Recent Examples and Considerations

Clinical Pharmacology &Therapeutics, Volume 117, Issue 4, Page 910-919, April 2025.
Recent discussions about the utilization of real‐world data (RWD) and real‐world evidence (RWE) have been more focused on drug development for regulatory approval rather than during the post‐marketing stage. In Japan, RWD/RWE have been practically utilized as an external control for drug approval.
Junichi Asano, Hiromi Sugano, Hiroyuki Murakami, Atsushi Noguchi, Yuki Ando, Yoshiaki Uyama   +5 more
wiley   +1 more source

FDA's stamp of approval: Unveiling peptide breakthroughs in cardiovascular diseases, ACE, HIV, CNS, and beyond

Journal of Peptide Science, Volume 30, Issue 11, November 2024.
This review provides an overview of FDA‐approved peptides, particularly those targeting cardiovascular diseases, human immunodeficiency, and central nervous system diseases, and also addresses conditions such as osteoporosis, thrombocytopenia, Cushing's disease, and hypoglycemia.
Othman Al Musaimi
wiley   +1 more source

Vosoritide therapy in Brazil: Consensus on the Diagnosis, Monitoring, and Management of Achondroplasia

Background Achondroplasia is the most common non-lethal disproportionate skeletal dysplasia and is associated with significant lifelong medical, functional, and psychosocial complications.
Ivana van der Linden Nader, Louise Cominato, Juliana Lopes Yamin, Paula Frassinetti Vasconcelos de Medeiros, Paulo Ferrez Collett-Solberg, Juan Clinton Llerena Jr, Erlane Marques Ribeiro, Genoir Simoni, Guido de Paula Colares Neto, Julienne Angela Ramires de Carvalho, Rodrigo Ambrosio Fock, Lena Stilianidi Garcia, Ana Paula Bordallo, Wagner Antonio da Rosa Baratela, Têmis Maria Félix, Luiz Claudio Gonçalves de Castro, Ricardo Fernando Arrais, Crésio de Aragão Dantas Alves, Kênia Maria de Souza Rio   +18 more
core   +1 more source

Clinical utility of comprehensive gene panel testing for common and rare causes of skeletal dysplasia and other skeletal disorders: Results from the largest cohort to date

American Journal of Medical Genetics Part A, Volume 194, Issue 9, September 2024.
Abstract Molecular genetics enables more precise diagnoses of skeletal dysplasia and other skeletal disorders (SDs). We investigated the clinical utility of multigene panel testing for 5011 unrelated individuals with SD in the United States (December 2019–April 2022).
Gretchen MacCarrick, Swaroop Aradhya, Mitch Bailey, Dorna Chu, Abigail Hunt, Emanuela Izzo, Deborah Krakow, William Mackenzie, Sarah Poll, Cathleen Raggio, Renée Shediac, Klane K. White, Heather M. McLaughlin, Guillermo Seratti   +13 more
wiley   +1 more source

Clinical outcomes and medical management of achondroplasia in Japanese children: A retrospective medical record review of clinical data

American Journal of Medical Genetics Part A, Volume 194, Issue 8, August 2024.
Abstract Achondroplasia (ACH) is a rare, autosomal dominant skeletal dysplasia characterized by short stature, characteristic facial configuration, and trident hands. Before vosoritide approval in Japan, patients with ACH could start growth hormone (GH) treatment at age 3 years.
Hiroyuki Saitou, Taichi Kitaoka, Takuo Kubota, Junko Kanno, Hiroshi Mochizuki, Toshimi Michigami, Kosei Hasegawa, Ikuma Fujiwara, Takashi Hamajima, Daisuke Harada, Yuko Seki, Keisuke Nagasaki, Sumito Dateki, Noriyuki Namba, Hirofumi Tokuoka, Jeanne M. Pimenta, Shelda Cohen, Keiichi Ozono   +17 more
wiley   +1 more source

Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study

CPT: Pharmacometrics &Systems Pharmacology, Volume 13, Issue 7, Page 1103-1116, July 2024.
Abstract While randomized, placebo‐controlled, double‐blinded clinical studies are the gold standard for evaluating the efficacy of investigational drugs, the use of placebo in children with achondroplasia should be limited because it provides no clinical benefit while exhausting study participants' treatment window.
Wenlian Qiao, Martin Boucher, Alison Slade, Vikas K. Dawra   +3 more
wiley   +1 more source

CHANGING THE TRAJECTORY OF GROWTH: THE APPLICATION OF VOSORITIDE IN CHILDREN WITH ACHONDROPLASIA. A CHANCE FOR IMPROVED QUALITY OF LIFE? [PDF]

Achondroplasia (ACH) is the most common skeletal dysplasia and the leading genetic cause of dwarfism. It is characterized by disproportionate short stature and lifelong medical complications, with an estimated incidence of 1 in 25,000–30,000 live births.
Marceli Mesyasz, Michalina Zagalska, Tomasz Tamborski, Emilia Gabriela Chrzanowska, Adam Jan Ząbek, Kamil Kerknawi, Jakub Paweł Kuźniar, Julia Szlążek, Paulina Mikulec, Marta Turek   +9 more
core   +1 more source