PUBLISHER: DataM Intelligence | PRODUCT CODE: 1345435
PUBLISHER: DataM Intelligence | PRODUCT CODE: 1345435
Hermansky-Pudlak syndrome (HPS) is caused by homozygous or compound heterozygous mutations in 1 of 11 genes that encode components in one of four protein complexes: adapter protein 3 (AP-3) and biogenesis of lysosome-related organelles complex 1, 2, and 3 (BLOC-1, BLOC-2, and BLOC-3). All four complexes support intracellular biogenesis and trafficking of lysosome and lysosome-related organelles (LROS). LROS include melanosomes, platelet dense bodies (also called delta granules), lamellar bodies of type II pneumocytes, and granule proteins of cytotoxic and suppressor T cells and natural killer (NK) cells.
Patients with HPS have a bleeding tendency associated with poor platelet aggregation. Platelets in patients with the syndrome have abnormal aggregation with collagen, thrombin, epinephrine, and adenosine diphosphate (ADP).
Furthermore, the Hermansky pudlak syndrome market is driven by various factors the vast consumer base increased awareness of bleeding problems and accessible remedies, and advantageous compensation policies drive the industry have significant key players like Ferring Pharmaceuticals, Dr. Reddy's, Sagent Pharma, Ubi Pharm and others.
The rise in the prevalence of rare diseases is one of the key factors that help the market during the forecast period. Rare diseases are present across the medical spectrum. Some are widely recognized by name, such as cystic fibrosis, while others are less known, such as cat eye syndrome. Most cancers (all but a few types) are rare. There are rare neurological and neuromuscular diseases, metabolic diseases, chromosomal disorders, skin diseases, bone and skeletal disorders, and rare diseases affecting the heart, blood, lungs, kidneys, and other body organs and systems.
For instance, according to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. This equates to 1 in 10 Americans, or one on every elevator and four on every bus.
The rise in the prevalence of Hermansky Pudlak syndrome is one of the key factors that help the market to grow during the forecast period. For instance, according to an updated article published at NCBI Hermansky-Pudlak syndrome (HPS) is rare, with a prevalence of 1 to 9 in 1,000,000 individuals worldwide but is more common in certain populations. As an example, the prevalence is 1 in 1800 individuals in the northwestern region of Puerto Rico, where approximately 1 in 22 individuals are carriers of the mutation. Most HPS type 1-affected individuals in Puerto Rico have the same founder 16-base-pair duplication in exon 15 of HPS1.
The failure of national organizations and other institutions in poor countries to raise awareness about certain ailments resulted in low testing penetration. This issue stifles market growth by hindering early diagnosis and appropriate patient care. Patients with HPS should obtain a preoperative pulmonary evaluation. The anesthesia team should be aware of bleeding tendencies and potential pulmonary complications as part of the syndrome. Patients with HPS may have pulmonary complications following general anaesthesia. Further, patients with the syndrome need careful post-operative monitoring.
The global Hermansky Pudlak Syndrome market is segmented based on type, therapy type, route of administration, distribution channel and region.
The desmopressin segment from the therapy type segment accounted for 41.7% and it is expected to be dominated during the The effectiveness of desmopressin acetate in HPS would likely depend on the individual's specific bleeding symptoms and the extent of platelet dysfunction. In some cases, it might provide modest relief for certain bleeding issues.
Excessive bleeding in individuals with HPS may be treated with medications that promote blood coagulation such as desmopressin (DDAVP), which has been shown to be effective for some patients. Other agents include aminocaproic acid, tranexamic acid and recombinant factor seven. For more serious bleeding, platelet transfusions can also be used and if possible, tissue-matched platelets are recommended.
North America is estimated to hold about 38.4 % of the total market share throughout the forecast period, due to its cost-effectiveness and access to healthcare facilities, improved treatment options, Patient awareness of bleeding disorders, as well as private-sector and government Therapeutics initiatives, assisting in market development. Also due to the preliminary stage acceptance of proven medical technology, the market in the area is the rapid ongoing expansion of firms operating in the drug sector.
For instance, in April 2023, Biopharma company Ocean Biomedical, Inc. announced its commitment to developing viable treatment options for Hermansky-Pudlak Syndrome (HPS). The company disclosed its plans to target OCF-203, its pulmonary fibrosis treatment candidate, as a therapeutic for fatal pulmonary fibrotic conditions caused by HPS.
Ocean's anti-fibrosis platform aims to target significant unmet needs for conditions like Idiopathic Pulmonary Fibrosis (IPF) and Hermansky-Pudlak Syndrome, and it has the potential to expand further into other areas like scleroderma, alcoholic liver disease, NASH, and kidney failure.
The major global players in the market include: Ferring Pharmaceuticals, Dr Reddy's, Sagent Pharma, Ubi Pharm, Amring Pharmaceuticals, Avet Pharmaceuticals, McKesson, Hybio Pharmaceuticals, Midas Pharma, SG Pharma and among others.
The overall impact of COVID-19 had a substantial impact on the global Hermansky Pudlak syndrome market, as this syndrome is a rare disease that requires specialized medical care and treatment options but COVID-19 has disrupted the overall healthcare systems in which people having Hermansky Pudlak syndrome treatment options became unaffordable which indeed impacted the overall market.
The Global Hermansky Pudlak Syndrome Market report would provide approximately 61 tables, 58 figures and 186 Pages.
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