Last update 19 Sep 2024

Hydranencephaly With Renal Aplasia-Dysplasia

Last update 19 Sep 2024

Basic Info

Synonyms

HYDRANENCEPHALY WITH RENAL APLASIA-DYSPLASIA, Hydranencephaly with Renal Aplasia-Dysplasia, MARCH

+ [6]

Introduction

A rare genetic lethal multiple congenital anomalies/dysmorphic syndrome with characteristics of severe hydranencephaly and renal dysplasia or agenesis. Pregnancy is complicated by oligo or anhydramnios, leading to features of Potter sequence (including typical facies and microretrognathia, limb contractures, talipes equinovarus, and pulmonary hypoplasia) in the fetus. Affected fetuses either die in utero or shortly after birth. Histology of the brain shows widespread presence of multinucleated neurons and glial cells.

Clinical Trials associated with Hydranencephaly With Renal Aplasia-Dysplasia

NCT01891916 / Unknown statusNot ApplicableIIT

Preventive Effect of Lactobacillus GG on Atopic Manifestation in Children With Cow's Milk Allergy

Food allergy (FA), defined as an adverse immune response to food allergens, is among the most frequent allergic disorders in childhood and it has recognized as a major paediatric health problem due to the severity of the reactions and the dramatic increase over the past decades. Cow's milk allergy (CMA) is the most frequent FA in children worldwide, and it has been demonstrated that it could be the first manifestation of the so-called "atopic march", characterized by the occurrence of other allergic disorders in the subsequent years after the onset of CMA. In a previous study, involving children with CMA over a period of 5 years, 40% developed asthma, 21% atopic eczema, and 43% allergic rhinitis. Similar results have been reported in a recent study on Finnish children Intestinal microflora appears to have a crucial role in the development of atopic disorders. Children with atopic diseases have different commensal bacterial groups in the gut compared to non-atopic children, and differences are also found between countries with high and low incidence of atopic diseases. There is currently great interest in manipulating the normal microbiota to accrue health benefits through an approach known as "probiotics." Probiotics are defined as "live microorganisms which when administered in adequate amounts confer a health benefit on the host". The conceptual basis of possible use of probiotics in the prevention and treatment of atopic disorders is well grounded. Lactobacillus GG (LGG) is the most studied probiotic in the prevention and treatment of atopic disorders. Wide and well-designed clinical studies have provided several evidences on the efficacy of LGG as preventive or therapeutic strategy in pediatric atopic disorders. More recently, in vitro studies have provided evidences on the potent immunoregulatory role and on the influence on intestinal microflora composition (toward a more beneficial composition in the prevention and treatment of atopic disorders) elicited by LGG. This view has been further reinforced by recent research showing that LGG is able to improve recovery of intestinal symptoms in infants with CMA-induced allergic colitis.

Start Date01 Oct 2008

Sponsor / Collaborator

Università degli Studi di Napoli Federico II

100 Clinical Results associated with Hydranencephaly With Renal Aplasia-Dysplasia

100 Translational Medicine associated with Hydranencephaly With Renal Aplasia-Dysplasia

0 Patents (Medical) associated with Hydranencephaly With Renal Aplasia-Dysplasia

265

Literatures (Medical) associated with Hydranencephaly With Renal Aplasia-Dysplasia

01 Jan 2024·Allergologia et immunopathologia

Resveratrol modulates the Nrf2/NF-κB pathway and inhibits TSLP-mediated atopic march.

Article

Author: Quan He ; Weihua Liu ; Zi Chen ; Guangfei Wei ; Jingxian Jiang ; Liuchao Zhang ; Linfu Zhou

BACKGROUND:

Resveratrol has been found to have anti-inflammatory and anti-allergic properties. The effects of resveratrol on thymic stromal lymphopoietin (TSLP)-mediated atopic march remain unclear.

PURPOSE:

To explore the potential role of resveratrol in TSLP-mediated atopic march.

METHODS:

The atopic march mouse model was established by topical application of MC903 (a vitamin D3 analog). Following the treatment with resveratrol, airway resistance in mice was discovered by pulmonary function apparatus, and the number of total cells, neutrophils, and eosinophils in bronchoalveolar lavage fluid was counted. The histopathological features of pulmonary and ear skin tissues, inflammation, and cell infiltration were determined by hematoxylin and eosin staining. The messenger RNA (mRNA) levels of TSLP, immunoglobulin E, interleukin (IL)-4, IL-5, and IL-13 were measured by real-time quantitative polymerase chain reaction. The protein expression of nuclear factor kappa B (NF-κB)/nuclear factor erythroid 2-related factor 2 (Nrf2) signaling-associated molecules (p-p65, p65, p-I kappa B kinase alpha (IκBα), IκBα, Nrf2, and TSLP) in lung and ear skin tissues were assessed by Western blot analysis.

RESULTS:

Resveratrol attenuated airway resistance and infiltration of total cells, eosinophils, and neutrophils in both lung and ear skin tissues. Resveratrol ameliorates serum inflammatory markers in allergic mice. Moreover, the phosphorylation levels of NF-κB pathway-related proteins were significantly reduced by administration of resveratrol in allergic lung and ear skin tissues. Similarly, the protein expression of TSLP in both lung and ear skin tissues was reduced by resveratrol, and Nrf2, a protector molecule, was increased with resveratrol treatment.

CONCLUSION:

Resveratrol attenuates TSLP-reduced atopic march through ameliorating inflammation and cell infiltration in pulmonary and ear skin tissues by inhibiting the abnormal activation of NF-κB signaling pathway.

01 Jan 2024·JID innovations : skin science from molecules to population health

Antigen Protease Activity on Intact or Tape-Stripped Skin Induces Acute Itch and T Helper Sensitization Leading to Airway Eosinophilia in Mice.

Article

Author: Toru Kimitsu ; Seiji Kamijo ; Tomoko Yoshimura ; Yurie Masutani ; Saya Shimizu ; Keiko Takada ; Punyada Suchiva ; Hideoki Ogawa ; Ko Okumura ; Shigaku Ikeda ; Toshiro Takai

Respiratory allergen sources such as house dust mites frequently contain proteases. In this study, we demonstrated that the epicutaneous application of a model protease antigen, papain, onto intact or tape-stripped ear skin of mice induced acute scratching behaviors and T helper (Th)2, Th9, Th17/Th22, and/or Th1 sensitization in a protease activity-dependent manner. The protease activity of papain applied onto the skin was also essential for subsequent airway eosinophilia induced by an intranasal challenge with low-dose papain. With tape stripping, papain-treated mice showed barrier dysfunction, the accelerated onset of acute scratching behaviors, and attenuated Th17/Th22 sensitization. In contrast, the protease activity of inhaled papain partially or critically contributed to airway atopic march responses in mice sensitized through intact or tape-stripped skin, respectively. These results indicated that papain protease activity on epicutaneous application through intact skin or skin with mechanical barrier damage is critical to the sensitization phase responses, including acute itch and Th sensitization and progression to the airway atopic march, whereas dependency on the protease activity of inhaled papain in the atopic march differs by the condition of the sensitized skin area. This study suggests that exogenous protease-dependent epicutaneous mechanisms are a target for controlling allergic sensitization and progression to the atopic march.

22 Dec 2023·Medicina (Kaunas, Lithuania)

Atopic March or Atopic Multimorbidity-Overview of Current Research.

Review

Author: Iva Mrkić Kobal ; Davor Plavec ; Željka Vlašić Lončarić ; Ivana Jerković ; Mirjana Turkalj

The atopic march encompasses a sequence of allergic conditions, including atopic dermatitis, food allergy, allergic rhinitis, and asthma, that frequently develop in a sequential pattern within the same individual. It was introduced as a conceptual framework aimed at elucidating the developmental trajectory of allergic conditions during childhood. Following the introduction of this concept, it was initially believed that the atopic march represented the sole and definitive trajectory of the development of allergic diseases. However, this perspective evolved with the emergence of new longitudinal studies, which revealed that the evolution of allergic diseases is far more intricate. It involves numerous immunological pathological mechanisms and may not align entirely with the traditional concept of the atopic march. The objective of our review is to portray the atopic march alongside other patterns in the development of childhood allergic diseases, with a specific emphasis on the potential for a personalized approach to the prevention, diagnosis, and treatment of atopic conditions.

News (Medical) associated with Hydranencephaly With Renal Aplasia-Dysplasia

05 Jan 2021

·www.biospace.com

Antengene Announces Submission of IND Application in China for a Global Phase 3 Trial of ATG-010 (Selinexor) in Advanced or Recurrent Endometrial Cancer

SHANGHAI and HONG KONG, Jan. 4, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in class therapeutics in hematology and oncology, announced that it submitted an Investigational New Drug (IND) application to the National Medical Products Administration (NMPA) for ATG-010 (selinexor) in the treatment of Endometrial Cancer. This is a global Phase 3, multicenter, randomized, double-blind trial (SIENDO) aiming to evaluate the efficacy of ATG-010 compared to placebo as maintenance therapy in patients with advanced or recurrent endometrial cancer after combination chemotherapy and is being conducted at approximately 80 investigative sites around the world, including North America, Europe and Asia. Endometrial cancer is the most common cancer of the female reproductive tract and for patients who progress following initial chemotherapy there are limited treatment options with poor prognosis. The highest incidence rates are reported in developed countries and some economically developed areas of China. In recent years, with an increase in the prevalence of obesity, diabetes and hypertension, the incidence and mortality rate of endometrial cancer has been increasing with a trend towards younger women. ATG-010 is a first-in-class and only-in-class oral selective inhibitor of nuclear export, developed by Antengene and Karyopharm Therapeutics Inc. (NASDAQ: KPTI). It has been approved by the US Food and Drug Administration (FDA) for use in both multiple myeloma and diffuse large B-cell lymphoma, which are two major indications in hematological malignancies. In addition, the Phase 3 SIENDO trial of ATG-010 in patients with endometrial cancer has passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modification. "The positive data of the Phase 3 SEAL trial in liposarcoma and the ongoing Phase 3 SIENDO trial in endometrial cancer which just passed the interim analysis have suggested substantial potential of ATG-010 across multiple solid tumors. Filing an IND with the NMPA in China is an important step in the clinical development of ATG-010 as we expect to explore additional therapeutic areas of our novel oral drug candidate," said Dr. Jay Mei, Founder, Chairman and CEO of Antengene. "We look forward to initiating the Phase 3 global trial in patients with advanced or recurrent endometrial cancer in China, to provide Chinese patients with better treatment options." Professor Qi Zhou from the Gynecological Oncology Center of Chongqing University Cancer Hospital, the leading clinical investigator, said: "R&D capability in China has improved significantly in recent years. There are more innovative anti-tumor drugs moving towards clinical research and commercialization, and they are widely used in clinical practice. The emergence of targeted and immunotherapy drugs effectively improved the prognosis of patients. I look forward to advancing the research of ATG-010, a first-in-class selective nuclear export inhibitor, with Antengene, to improve the effect and increase the accessibility of treatment, so that patients can benefit from clinical results as soon as possible." About ATG-010 (selinexor, XPOVIO®) ATG-010 (selinexor, XPOVIO®), a first-in-class and only-in-class oral selective inhibitor of nuclear export compound discovered and developed by Karyopharm, is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including the Greater China, South Korea, Australia, New Zealand and the ASEAN countries. In July 2019, the US Food and Drug Administration (FDA) approved ATG-010 in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (rrMM) and in June 2020 approved ATG-010 as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL). A Marketing Authorization Application (MAA) has also been submitted to the European Medicines Agency (EMA) with a request for conditional approval of ATG-010 in this same rrMM indication. On December 18, 2020, the supplemental New Drug Application (sNDA) requesting an expansion of its indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA. ATG-010 is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. ATG-010 is also being evaluated in several other mid-and later-Phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene's partner, Karyopharm, presented positive results from the Phase 3 randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral ATG-010 versus matching placebo in patients with liposarcoma. Karyopharm also recently announced that the ongoing Phase 3 SIENDO trial of ATG-010 in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021. Antengene is conducting two registrational Phase 2 clinical trials of ATG-010 in China for relapsed/refractory multiple myeloma (MARCH) and for relapsed/refractory diffuse large B-cell lymphoma (SEARCH), and has initiated clinical trials for high prevalence cancer types in the Asia Pacific region including peripheral T-cell lymphoma and NK/T-cell lymphoma (TOUCH) and KRAS-mutant non-small cell lung cancer (TRUMP). About Antengene Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage Asia-Pacific biopharmaceutical company focused on innovative oncology medicines. Antengene aims to provide the most advanced anti-cancer drugs to patients in China, the Asia Pacific Region and around the world. Since its establishment, Antengene has built a pipeline of 12 clinical and pre-clinical stage assets and obtained 11 investigational new drug approvals in Asia Pacific. The vision of Antengene is to "Treat Patients Beyond Borders". Antengene aims to address significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics. Forward-looking statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. * XPOVIO® is a registered trademark of Karyopharm Therapeutics Inc.. Company Codes: HongKong:6996

First in ClassImmunotherapy

04 Jan 2021

·www.biospace.com

Antengene Submits NDA for ATG-010 (Selinexor) in South Korea for rrMM and rrDLBCL

SHANGHAI and HONG KONG, Jan. 3, 2021 /PRNewswire/ -- Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in class therapeutics in hematology and oncology, announced that it has submitted a New Drug Application (NDA) with Orphan Drug Designation (ODD) to the South Korean Ministry of Food and Drug Safety (MFDS) for ATG-010 (selinexor, XPOVIO®) in combination with low dose dexamethasone for the treatment of adult patients with relapsed/refractory multiple myeloma (rrMM) and for ATG-010 (selinexor, XPOVIO®) as monotherapy to treat adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL). ATG-010 (selinexor, XPOVIO®) was granted orphan drug designation in South Korea in October 2020 and our US partner Karyopharm received U.S. Food and Drug Administration (FDA) approval for the treatment of patients with multiple myeloma after at least one prior therapy on December 18, 2020. The NDA submission includes positive data from the pivotal STORM and SADAL studies, which both demonstrated significant and meaningful efficacy with a manageable safety pro ATG-010 (selinexor, XPOVIO®). The STORM study is a Phase 2b, open-label, single-arm study evaluating ATG-010 (selinexor, XPOVIO®) plus low-dose dexamethasone in patients with rrMM who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody. The SADAL study is a Phase 2b, open label study evaluating ATG-010 (selinexor, XPOVIO®) in patients with rrDLBCL, not otherwise specified, including DLBCL arising from follicular lymphoma, who have received at least two prior therapies. ATG-010 (selinexor, XPOVIO®) is a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE) and it is the first drug approved by the FDA for use in both multiple myeloma and diffuse large B-cell lymphoma. In December 2020, the National Comprehensive Cancer Network (NCCN®) added three different triplet ATG-010 (selinexor, XPOVIO®) combination regimens, including SVd (selinexor, bortezomib and dexamethasone), SPd (selinexor, pomalidomide and dexamethasone) and SDd (selinexor, daratumumab and dexamethasone) to its Clinical Practice Guidelines in Oncology (NCCN® Guidelines) for previously treated multiple myeloma in the US. Antengene has submitted NDAs to the Health Sciences Authority (HSA) of Singapore and to the Australian Therapeutic Goods Administration (TGA) for ATG-010 in patients with rrMM and in patients with rrDLBCL. "The incidence of blood cancers increases with age, yet many healthcare providers still do not have sufficient innovative therapies available to help people with hematological malignancies, such as rrMM and rrDLBCL," Dr. Jay Mei, Founder, Chairman and CEO of Antengene said, "We are excited about the regulatory filing of ATG-010 in APAC markets, including South Korea. We believe that oral ATG-010 will expand and improve treatment options for hematological malignancies and therefore presents a significant advancement for patients requiring treatment of these life threatening diseases." About ATG-010 (selinexor, XPOVIO®) ATG-010 (selinexor, XPOVIO®), a first-in-class and only-in-class oral selective inhibitor of nuclear export compound discovered and developed by Karyopharm Therapeutics Inc. (NASDAQ: KPTI), is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including the Greater China, South Korea, Australia, New Zealand and the ASEAN countries. In July 2019, the US Food and Drug Administration (FDA) approved selinexor (XPOVIO®) in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (rrMM) and in June 2020 approved selinexor (XPOVIO®) as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL). A Marketing Authorization Application (MAA) has also been submitted to the European Medicines Agency (EMA) with a request for conditional approval of selinexor (XPOVIO®) in this same rrMM indication. On December 18, 2020, the supplemental New Drug Application (sNDA) with a request for an expansion of its indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA. Selinexor (XPOVIO®) is so far the first and only oral SINE compound approved by the FDA and is the first drug approved by the FDA for the treatment of both MM and DLBCL. Selinexor (XPOVIO®) is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene's partner, Karyopharm, presented positive results from the Phase 3 randomized, double blind, placebo controlled, cross-over SEAL study evaluating single agent, oral selinexor (XPOVIO®) versus matching placebo in patients with liposarcoma. Karyopharm also recently announced that the ongoing Phase 3 SIENDO study of selinexor (XPOVIO®) in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the study should proceed as planned without any modifications. Top-line SIENDO study results are expected in the second half of 2021. Antengene is conducting two registrational Phase 2 clinical trials of ATG-010 (selinexor, XPOVIO®) in China for relapsed/refractory multiple myeloma (MARCH) and for relapsed/refractory diffuse large B-cell lymphoma (SEARCH), and has initiated clinical trials for high prevalence cancer types in the Asia Pacific region including peripheral T-cell lymphoma and NK/T-cell lymphoma (TOUCH) and KRAS-mutant non-small cell lung cancer (TRUMP). About Antengene Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading clinical-stage Asia-Pacific biopharmaceutical company focused on innovative oncology medicines. Antengene aims to provide the most advanced anti-cancer drugs to patients in China, the Asia Pacific Region and around the world. Since its establishment, Antengene has built a pipeline of 12 clinical and pre-clinical stage assets and obtained 11 investigational new drug approvals in Asia Pacific. The vision of Antengene is to "Treat Patients Beyond Borders". Antengene aims to address significant unmet medical needs by discovering, developing and commercializing first-in-class/best-in-class therapeutics. Forward-looking statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. * XPOVIO® is a registered trademark of Karyopharm Therapeutics Inc.; NCCN® is a registered trademark of National Comprehensive Cancer Network. View original content: SOURCE Antengene Corporation Limited Company Codes: HongKong:6996, NASDAQ-NMS:KPTI

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Hydranencephaly With Renal Aplasia-Dysplasia

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