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Novel ACRV1/ALK2 Inhibitors and Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
Case ID:
TAB-3598
Web Published:
12/6/2022
Description:
This technology includes the identification and use of novel ACRV1/ALK2 inhibitors for the treatment of fibrodysplasia ossificans progressiva (FOP), an autosomal-dominant rare disease that affects one person in every 1-2 million. FOP is characterized by malformation of the great (big) toes during embryonic development and by progressive heterotopic endochondral ossification (HEO) postnatally, which leads to the formation of a second skeleton of heterotopic bone. Individuals with the classical features of FOP have the identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.
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Direct Link:
https://nih.technologypublisher.com/tech?title=Novel_ACRV1%2fALK2_Inhibitors_ and_Methods_for_Inhibiting_BMP_Signaling_for_the_Treatment_of_Fibrodysplasia _Ossificans_Progressiva_(FOP)
Category(s):
Endocrinology
Therapeutics
Research Materials
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For Information, Contact:
Suryanarayana Vepa
Technology Licensing Specialist
NIH Technology Transfer
301-827-7181
sury.vepa@nih.gov
Inventors:
Jian-kang Jiang
Khalida Shamim
Philip Sanderson
Wei Zheng
Xiuli Huang
Gregory Tawa
Asaf Alimardanov
Arthur Lee
Junfeng Huang
Wenwei Huang
Keywords:
ALK2
BMP
INHIBITING
Inhibitors
Methods
Novel
SIGNALING
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