MedPath

Idursulfase beta

Generic Name
Idursulfase beta
Brand Names
-
Drug Type
Biotech
Chemical Formula
-
CAS Number
1271734-34-9
Unique Ingredient Identifier
5GCF8EPY6R
Background

Idursulfase beta is under investigation in clinical trial NCT01645189 (Safety and Efficacy of Hunterase).

Indication

用于确诊的黏多糖贮积症Ⅱ型(MPSⅡ, 亨特综合征,属于罕见病)患者的酶替代治疗。

亨特综合征(黏多糖贮积症Ⅱ型,MPS II)是一种遗传性溶酶体贮积症(LSD),主要发生在男孩身上。由2-硫酸酯酶缺乏引起,2-硫酸酯酶是分解体内称为糖胺聚糖(GAGs)所需要的一种酶。没有这种酶,GAG就会累积并引发严重的骨骼、组织、神经和多器官并发症,并最终导致死亡。患病率为1.3/10万新生男孩。

Associated Conditions
-
Associated Therapies
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Denali Therapeutics Initiated with an Outperform at Baird

Baird rates Denali Therapeutics (DNLI) Outperform with a $31 target, predicting FDA approval for tividenofusp alfa by late 2025/early 2026. DNL310, targeting Hunter syndrome, may capture significant market share from Elaprase. Denali's Alzheimer’s and Parkinson’s programs are seen as promising long-term opportunities.
globenewswire.com
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Global Mucopolysaccharidosis Treatment Market to Witness Growth

The global mucopolysaccharidosis treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth is driven by advancements in biotechnology, enhanced diagnostic methods, increased awareness, and the availability of enzyme replacement therapy. Clinical trials for innovative treatments are expanding, despite high costs and lack of reimbursement policies limiting market growth.
finance.yahoo.com
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Global Mucopolysaccharidosis Treatment Market to Witness Robust Growth, Projected to Reach USD 5,014.5 Million by 2035 Growing at a CAGR of 5.9%

The global mucopolysaccharidosis (MPS) treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth drivers include advancements in biotechnology, enhanced diagnostic methods, increased awareness of rare diseases, and the availability of enzyme replacement therapy (ERT). Clinical trials and new drug approvals, such as Lysogene's LYS-SAF302 and JCR Pharmaceuticals' JR-141, are pivotal. Despite high treatment costs and reimbursement challenges, the market is set to expand, with ERT remaining the preferred treatment.
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