China
Africa
Explore chapters and articles related to this topic
Fibromyalgia Syndrome: Canadian Clinical Working Case Definition, Diagnostic and Treatment Protocols–A Consensus Document
Published in I. Jon Russell, The Fibromyalgia Syndrome: A Clinical Case Definition for Practitioners, 2020
Anil Kumar Jain, Bruce M. Carruthers, Maijorie I. van de Sande, Stephen R. Barron, C. C. Stuart Donaldson, James V. Dunne, Emerson Gingrich, Dan S. Heffez, Y.-K. Frances Leung, Daniel G. Malone, Thomas J. Romano, I. Jon Russell, David Saul, Donald G. Seibel
Growth hormone treatment: It is of great interest that administration of growth hormone to people with FMS reduces many of the symptoms associated with this disorder (354). It appears that response to human growth hormone [HGH] takes time since the treated subjects did not exhibit significant improvement until about three months of continuous therapy. Withdrawal of the drug usually resulted in recurrence of the symptoms. The dosage must be carefully monitored by measuring the insulin-like growth hormone-1 [IGF1] levels to avoid the development of gigantism and other potentially serious side effects. Regular injection therapy with this hormone is not universally appealing to FMS patients although it is remarkable how well they adapted to its use in the research study. To date, the cost of such therapy is too expensive [approximately US $ 1,000.00/month] to allow its widespread clinical use despite the fact that a HGH and IGF1 deficiency is demonstrable and administration of HGH is effective against at least some of the FMS symptoms.
Contaminated blood
Published in Mélinée Kazarian, Criminalising Medical Malpractice, 2020
Following the blood scandal, other healthcare scandals in France gave rise to the use of tromperie. For instance, in the human growth hormone scandal, doctors were prosecuted for tromperie and homicide involontaire for having prescribed and supplied human growth hormone contaminated with vCJD204 to patients.205 Similarly, the heads of two French laboratories which supplied anti-hepatitis B vaccine were prosecuted for tromperie for having supplied deficient vaccines to patients.206
Genetics of Endocrine Disorders and Diabetes Mellitus
Published in George H. Gass, Harold M. Kaplan, Handbook of Endocrinology, 2020
Bess Adkins Marshall, Abby Solomon Hollander
Newborns with growth hormone deficiency usually have normal length and weight. They may present with hypoglycemia or prolonged hyperbilirubinemia.2 Boys will often have a micropenis.3 In children with GH deficiency, the facial bones develop slowly, leading to protrusion of the frontal bones and poor development of the bridge of the nose. Tooth eruption is delayed. The hair is thin and the nail growth is poor. The voice is often high-pitched. The linear growth is retarded, and the child will often appear mildly obese and cherubic.2 These features can be corrected via therapy with recombinant human growth hormone (hGH). The discovery of the GH gene locus on human chromosome 17 made possible the development of recombinant hGH. In the last several years, much has been elucidated about the control of GH gene expression. The remainder of this section will focus on growth hormone gene expression, its regulation by transcription factors, and clinical examples of abnormalities of these processes.
Emerging drug targets for achondroplasia
Published in Expert Opinion on Therapeutic Targets, 2022
Individuals with achondroplasia experience a variety of medical issues over their lifespan, ranging from an increased risk of sudden infant death before age 5 years, to sleep disordered breathing, to spinal curvature and symptoms related to narrowing of the foramen magnum with cervico-medullary compression in infancy, and spinal canal stenosis in adulthood that often require surgery to decompress [3,6]. Until very recently, treatment has predominantly been expectant and surgical. Treatment with recombinant human growth hormone has been used in children with ACH and is an approved therapy for this indication in Japan. However, children with ACH have normal serum growth hormone levels, and the beneficial effect of growth hormone therapy on final adult height in these children has not been demonstrated [7]. Similarly, limb-lengthening surgical procedures have been used in some countries to increase height, but this practice varies considerably worldwide depending on resources and cultural expectations and norms. Currently, there is no international consensus regarding if limb lengthening surgery should be performed in children with ACH or regarding the timing when such surgery, if undertaken, should occur [3].
Approval of biosimilars: a review of unsuccessful regulatory filings
Published in Expert Opinion on Biological Therapy, 2021
Anurag S. Rathore, Hemlata Chhabra, Ankita Bhargava
EMA was the first regulatory agency to start reviewing applications for biosimilars approval. To date, EMA has approved 64 biosimilars within the product classes of 1) human growth hormone, 2) granulocyte colony-stimulating factor, 3) erythropoiesis-stimulating agent, 4) insulin, 5) follicle-stimulating hormone (FSH), 6) parathyroid hormone, 7) tumor necrosis factor (TNF) inhibitor, and 8) monoclonal antibodies for use in the EU [11]. Thus, far nine biosimilar approvals have been withdrawn after approval, one biosimilar has been withdrawn before approval, and two biosimilar applications have been refused by the agency (Table 1). This leaves a total of 55 biosimilars authorized for use in Europe [12]. In spite of the patent expiration in EU for Erbitux (cetuximab) and Aranesp (darbepoetin alfa) in 2014 and 2016, respectively, no biosimilars have been approved for the same. A number of biosimilar products for cetuximab including ABP-494 (Actavis/Amgen) and CT-P15 (Celltrion) are in development [13]. Two products, Retacrit (Hospira) and Silapo (Stada), are being approved by EMA for similar indications such as Aranesp including anemia, chronic kidney failure, cancer, and autologous blood transfusion. The reference medicine for both the products is Eprex/Erypo, which contains epoetin alpha as an active substance. [14]. Other blockbuster drugs that are set to lose their patents in the coming 2 years in Europe include Soliris (2020), Benlysta (2021), Lemtrada (2021), Avastin (2022), and Lucentis (2022) [15].
Hypotonia and delayed motor development as an early presentation of Lowe syndrome: case report and literature review
Published in Acta Clinica Belgica, 2019
Sara David, Kathleen De Waele, Bram De Wilde, Franny Faes, Olivier Vanakker, Sophie Walraedt, Agnieszka Prytuła
As reported by Zaniew et al. patients with LS often require bicarbonate, citrate and vitamin D supplements, but in fact these are prescribed insufficiently [7]. The use of thiazides, indomethacin and ACE-inhibitors to correct acidosis and proteinuria is controversial because of their side-effects, which may put patients with LS into a risk of dehydration and AKI [7]. A small number of patients have successfully undergone renal transplantation [3]. Despite of good correction of the tubular dysfunction, some patients develop pathological fractures. Intravenous pamidronate treatment can be considered [3]. For growth impairment, recombinant human growth hormone therapy is only indicated in case of demonstrable growth hormone deficiency [2]. Tranexamic acid administration in times of bleeding risk or provocation usually ameliorates platelet function in LS [1].