Late onset congenital adrenal hyperplasia

Fabry Disease Industry Insights 2023-2032: Epidemiology, Drug Pipeline, Emerging Treatments, Market Trends, Expert Opinions - ResearchAndMarkets.com

Retrieved on: 
Friday, August 25, 2023
Health, Other Health, EU4, Subject-matter expert, Chiesi Farmaceutici, Internet, USD, AAV, Protalix BioTherapeutics, ERT, GLA, Sangamo Therapeutics, Growth, Late onset congenital adrenal hyperplasia, Classic, Enzyme replacement therapy, Epidemiology, PEGylation, SME, Pharmaceutical industry, Vaccine, Video game, Dentistry, Fabry, EU, Fabry disease

This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.

Key Points: 
  • This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.
  • The report provides a comprehensive understanding of Fabry Disease epidemiology, including historical and forecasted data.
  • The Fabry Disease market experienced 15,290 diagnosed prevalent cases in the 7MM in 2022, with 8,355 cases in the US alone.
  • The Fabry Disease market size is projected to increase at a CAGR of 6.3% from 2023 to 2032, driven by awareness and the launch of emerging drugs.

Ultragenyx Announces Additional Positive Multi-Year Durability Data from Phase 1/2 AAV Gene Therapy Studies

Retrieved on: 
Monday, November 29, 2021
Patient, Private Securities Litigation Reform Act, Tissue, Infant, Security (finance), Liver transplantation, Disease, Toxic leukoencephalopathy, Lactic acidosis, Biology, Diet, Hypoglycemia, Blood, U.S. Securities and Exchange Commission, Therapy, COVID-19, Glycogen, Ammonia, Orphan, Kidney, Investment, Risk, GLOBE, International Congress on Tuberculosis, Fast Track, Â, OTC, HIV disease progression rates, Fasting, Uncertainty, AAV, Late onset congenital adrenal hyperplasia, Ornithine transcarbamylase deficiency, NASDAQ, Glucose, Glycogen storage disease, Liver, Pharmaceutical industry, Medical imaging

Across all patients in the Phase 1/2 study, to date there have been no infusion-related adverse events, no treatment-related serious adverse events, and no dose-limiting toxicities reported.

Key Points: 
  • Across all patients in the Phase 1/2 study, to date there have been no infusion-related adverse events, no treatment-related serious adverse events, and no dose-limiting toxicities reported.
  • It is caused by a defective gene coding for the enzyme G6Pase-, resulting in the inability to regulate blood sugar (glucose).
  • DTX401 is an investigational adeno-associated virus (AAV) type 8 gene therapy designed to deliver stable expression and activity of G6Pase- under control of the native promoter.
  • DTX301 is an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion.

Spruce Biosciences Announces Publication of Phase 2 Results for Tildacerfont in Journal of Clinical Endocrinology and Metabolism

Retrieved on: 
Monday, June 21, 2021
Biotechnology, General Health, Health, Pharmaceutical, Clinical trials, Endocrine diseases, Organ systems, Endocrine system, Hyperandrogenism, Women's health, Polycystic ovary syndrome, Polycystic disease, Late onset congenital adrenal hyperplasia, Tildacerfont, Spruce Biosciences, TILDACERFONT, SPRUCE BIOSCIENCES

SPR001-201 and SPR001-202 comprised the entire Phase 2a clinical development program for tildacerfont in adult classic CAH.

Key Points: 
  • SPR001-201 and SPR001-202 comprised the entire Phase 2a clinical development program for tildacerfont in adult classic CAH.
  • Tildacerfont has been evaluated in 171 patients across seven clinical trials in which it has been generally well tolerated.
  • Spruce is also developing tildacerfont for women suffering from a rare form of polycystic ovary syndrome (PCOS) with primary adrenal androgen excess.
  • Such forward-looking statements include statements regarding, among other things, the results, conduct, progress and timing of Spruces clinical trials.

Crinetics Pharmaceuticals Provides New Data from Paltusotine and ACTH Antagonist Development Programs at European Congress of Endocrinology

Retrieved on: 
Friday, September 4, 2020
Endocrine diseases, Adrenal gland disorders, Organ systems, Endocrinology, RTT, Cushing's syndrome, Adrenocorticotropic hormone, Congenital adrenal hyperplasia, Acromegaly, Adrenal gland, Hyperplasia, Late onset congenital adrenal hyperplasia

Crinetics is developing an ACTH antagonist for the treatment of diseases associated with excess ACTH such as Cushings disease and congenital adrenal hyperplasia (CAH).

Key Points: 
  • Crinetics is developing an ACTH antagonist for the treatment of diseases associated with excess ACTH such as Cushings disease and congenital adrenal hyperplasia (CAH).
  • We look forward to achieving the next milestone in this program by advancing our lead ACTH antagonist candidate into Phase 1.
  • Adverse events associated with paltusotine were generally mild and transient and were consistent with those reported withother somatostatin agonists.
  • The results of this study suggest that paltusotine exhibits excellent properties appropriate for chronic once-daily oral treatment of patients with acromegaly.