Background Each rare disease only affects a small number of population. However, a total of 7000 rare diseases may affect 10% of the population. Due to the severity and lack of rare disease awareness, rare disease represents a huge challenge for the healthcare system. In Western countries, patient organizations have been playing an integral role in raising awareness, advocating legislation, and supporting drug development. This study aims to assess the unmet needs of rare disease patient organizations in China, and identify their unmet needs, providing essential information for the government and legislators. Results A total of 28 individuals representing 28 patient organizations in China were interviewed. Most organizations do not have official registration, employees, written standard operation protocol, or reliable financial resources. Misdiagnosis or delayed diagnosis is common, and treatment is often lacking. Due to the lack of financial resources, no organizations have been able to sponsor academic research, unlike their counterparts in Western countries. As to challenges, 71.4% of interviewees listed lack of rare disease awareness among the general public, while 67.9% selected lack of financial resources. Further, only 7.3% of these organizations received support from the government, and 28.6% received support from the general public. As to recommendations to the government, 82.1% of interviewees selected special insurance programs for rare diseases because rare diseases have been generally excluded from the national medical insurance programs. In addition, 78.6% of interviewees recommended to stimulate rare disease research, 75% recommended to import orphan drugs, and 71.4% recommended legislation of an orphan drug act, highlighting the urgent need of therapies. Conclusions Due to lack of support and rare disease awareness, patient organizations in China are still in the early phase. To empower these patient organizations, the interviewees’ recommendations, including legislating orphan drug act and releasing official definition of rare diseases, should be considered by the government and legislators.
We investigate encoding and decoding of two-dimensional information using the orbital angular momentum (OAM) of light. Spiral phase plates and phase-only spatial light modulators are used in encoding and decoding of OAM states, respectively. We show that off-axis points and spatial variables encoded with a given OAM state can be recovered through decoding with the corresponding complimentary OAM state.
In situ self-assembly has emerged as a powerful technology in preparation of nanoparticles owing to its solutionprocessing-free advantage and scalable production. This new technology allows us to facilely obtain a wide range of nanoobjects of architecture and dimension, including spheres, cylinders, vesicles, and tubes; however, it has limitation in the construction of sophisticated superstructures such as helix or superhelix. Here, we describe a new in situ self-assembly method, light-click in situ selfassembly, which can induce the concurrence of polymer coupling and self-assembly through photoclick chemistry. We use this technique to the direct synthesis of helical assemblies. Using two clickable molecules as the reactive precursors, their photoligation can synchronize with their self-assembly process and in situ fabricate ergodic helical nanostructures with chiral bias, evolving from nonhelical filaments to single-stranded helical fibrils to multistranded helical fibrils to complex superhelical fibers. Such helically hierarchical shape evolution is dependent on the photoreaction extent. Quantifying the relationship of reaction conversion and resulting morphology can establish a full phase diagram of helical self-assembly. Moreover, utilizing this strategy, one can on-demand pause and resume the assembly process by switching on and off the light stimulus in order to achieve temporal control of the helicity hierarchy.
Zero-shot cross-domain slot filling alleviates the data dependence in the case of data scarcity in the target domain, which has aroused extensive research. However, as most of the existing methods do not achieve effective knowledge transfer to the target domain, they just fit the distribution of the seen slot and show poor performance on unseen slot in the target domain. To solve this, we propose a novel approach based on prototypical contrastive learning with a dynamic label confusion strategy for zero-shot slot filling. The prototypical contrastive learning aims to reconstruct the semantic constraints of labels, and we introduce the label confusion strategy to establish the label dependence between the source domains and the target domain on-the-fly. Experimental results show that our model achieves significant improvement on the unseen slots, while also set new state-of-the-arts on slot filling task. 1
Fabricating nanoscale assemblies that can respond to gas signaling molecules has emerged as a field of growing interest owing to their unique biomedical applications in gas-guided delivery and gas therapeutics. Yet, among a variety of endogenous gaseous biosignals, exploiting sulfur dioxide (SO 2 ) as a cue for controllable self-assembly remains elusive, despite its crucial two-sided roles both in physiology and pathology. Here we show a SO 2 -responsive polymersome system assembled from a novel class of cyanine-containing block copolymers. By intake of SO 2 gas, the tautomerism of cyanine drives such vesicles to continuously deform, and change into long nanotubes through axial stretching and anisotropic extrusion of the membranes. Unexpectedly, during this order-to-order phase transition, their membranes manifest well SO 2 -dosedependent permselectivity, which allows the cargos of different sizes loaded therein to be selectively transferred across the bilayers. This study would inspire us to better understand and mimic the function of gas signaling molecules in shifting biomembrane shape and managing transmembrane traffic.
For the lack of accurate test results in design and maintenance of desert-crossing highways in the Tengger Desert of western China, the GDS true-triaxial system was used to conduct the drained test on dense sand. Under the condition of different intermediate principal stress ratio b-value, the results showed that the stress-strain relationships in three orthogonal directions had significant differences, presenting significant anisotropy. The peak of the generalized shear stress increased with the increase of b-value. Except under the condition of b = 0, the specimen contracted firstly and then dilated, while the others dilated. The results of the different confining pressures showed that the stress-strain relationships appeared as a hardening type at low confining pressures, and as the confining pressure increased, the stress-strain relationships exhibit hardening, peaking, softening, and stable deformation characteristics. At low confining pressure, the contractive behaviors were not obvious, mainly as dilatancy, and as the confining pressure increased, the dilatancy increased gradually. The specimen transformed contract to dilatancy, and when the confining pressure reached 800 kPa, the specimen exhibited contractive behavior. The test results will provide data for subgrade design and construction in desert area.
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