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Novel medicine could address short stature in children
12 Apr 2024 21:45 GMT
… hypochondroplasia, a genetic cause of short stature in children, achieved an … medicine that has been developed to specifically target the … trial will help support future trials of vosoritide as a treatment … how well tolerated the medication was and how some …
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Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positive
11 Apr 2024 16:32 GMT
Neurofibromatosis type 1 is an inherited disease in which the patient develops benign (non-cancerous) tumours along the nerves. The severity of the disease varies from patient to patient, and symptoms include pale, coffee-coloured patches, freckles in …
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First clinical trial of vosoritide for children with hypochondroplasia shows increased growth
11 Apr 2024 23:30 GMT
… first medicine that has been developed to specifically target the … -its-kind to treat children with genetic short stature who do not … the trial, the researchers also noted there were no treatment-related … see how well tolerated the medication was and how some …
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DSMB recommends continuation of phase 3 trial evaluating central precocious puberty treatment
03 Apr 2024 19:34 GMT
… the potential for significantly short stature in adulthood and associated social … -releasing hormone (GnRHa) treatment for CPP.2
Other inclusion … trial," said Bassem Elmankabadi, MD, senior vice president, clinical development, Foresee Pharmaceuticals …
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HARMONY BIOSCIENCES INITIATES GLOBAL PHASE 3 REGISTRATIONAL TRIAL (TEMPO STUDY) OF PITOLISANT IN PATIENTS WITH PRADER-WILLI SYNDROME
03 Apr 2024 12:21 GMT
… .S. and is FDA approved to treat EDS or cataplexy … features include low muscle tone, short stature, and cognitive impairment.
About … medication, is approved by the U.S. Food and Drug … we specialize in developing and delivering treatments for rare neurological …
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Foresee Pharma gets positive recommendation from DSMB to continue Casppian phase 3 study of Leuprolide injectable emulsion to treat CPP
02 Apr 2024 07:10 GMT
… that Foresee Pharmaceuticals continue the trial with a … with statistically significant treatment effects in adult … vice president of clinical development at Foresee Pharmaceuticals.
“Foresee… risk of having significantly short stature as adults in …
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18 Apr 2024 20:31 GMT
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Changes in FeNO, d-ROMs, and BH4 by Intravenous L-Arginine in Children and Its Putative Role in Asthma Treatment
19 Mar 2024 14:20 GMT
… for the diagnosis of short stature in Osaka City … and date of treatment (order of treatment) was adjusted. To … Inc, Takeda Pharmaceutical Co, Shionogi Pharma, and Janssen Pharmaceutical K.K … JM, Farriaux JP. Developmental aspects of pteridine metabolism …
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What are rare diseases that deter children's growth and their treatments?
14 Mar 2024 08:16 GMT
… new medications are being developed to prevent and treat stunting for … been the mainstay of treatment for short stature. However, in 2021 … Europe. The drug is an orphan drug developed based on … to introduce new drugs developed by foreign countries quickly …
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Factors Related to Treatment Non-Adherence Among Caregivers of Pediatric and Adolescent Growth Hormone Deficiency Patients in Japan
07 Mar 2024 17:39 GMT
… survey instrument was developed by the authors … consistent with Good Pharmacoepidemiology Practices and the … improve satisfaction with drug treatment and improve treatment management.
Study … on medication adherence in children with short stature receiving …