Crowdfunding is emerging as an alternative form of funding for medical purposes, with
capital being raised directly from a broader and more diverse audience of investors. In this …

Hypomyelinating leukodystrophies are heritable disorders defined by lack of development of
brain myelin, but the cellular mechanisms of hypomyelination are often poorly understood …

Cells rely heavily on microtubules for several processes, including cell division and
molecular trafficking. Mutations in the different tubulin-α and-β proteins that comprise …

Objective Hypomyelinating disorders are a group of clinically and genetically
heterogeneous diseases characterized by neurological deterioration with hypomyelination …

TUBB4A-associated disorder is a rare condition affecting the central nervous system. It
displays a wide phenotypic spectrum, ranging from isolated late-onset torsion dystonia to a …

The tubulin beta-4A gene (TUBB4A) is associated with two different clinical conditions,
dystonia type 4 (DYT4) and hypomyelination with atrophy of the basal ganglia and …

Finance has a great impact on real economy's development. From a historical perspective,
financial innovation is often coupled with technological advancement. Among various …

Background AnWj is a high‐incidence blood group antigen associated with three clinical
disorders: lymphoid malignancies, immunologic disorders, and autoimmune hemolytic …

In this study, we report a family with X-linked recessive syndrome caused by mutated
AMMECR1 and characterized by elliptocytosis with or without anemia, midface hypoplasia …

Over 300 million people are affected by about 7000 rare diseases globally. There are
tremendous resource limitations and challenges in driving research and drug development …