更新于：2024-05-01

Hereditary Neoplastic Syndromes

遗传性肿瘤综合征

更新于：2024-05-01

基本信息

别名

Cancer Syndrome, Hereditary、Cancer Syndromes, Hereditary、Familial Neoplastic Syndrome

+ [27]

简介

The condition of a pattern of malignancies within a family, but not every individual's necessarily having the same neoplasm. Characteristically the tumor tends to occur at an earlier than average age, individuals may have more than one primary tumor, the tumors may be multicentric, usually more than 25 percent of the individuals in direct lineal descent from the proband are affected, and the cancer predisposition in these families behaves as an autosomal dominant trait with about 60 percent penetrance.

关联

项与遗传性肿瘤综合征相关的药物

Tunlametinib

妥拉美替尼

靶点

MEK

作用机制

MEK1抑制剂 [+1]

在研机构

上海科州药物研发有限公司初创企业

原研机构

上海科州药物研发有限公司初创企业

在研适应症

NRAS突变黑色素瘤 [+9]

非在研适应症

KRAS突变非小细胞肺癌

最高研发阶段批准上市

首次获批国家/地区

中国

首次获批日期2024-03-12

Ganaxolone

加奈索酮

靶点

GABAA receptor

作用机制

GABAA receptor激动剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2022-03-18

Palovarotene

帕罗伐汀

靶点

RARγ

作用机制

RARγ激动剂

在研机构

Ipsen SA [+2]

原研机构

Clementia Pharmaceuticals, Inc.

在研适应症

骨化性肌炎

非在研适应症

干眼综合征 [+2]

最高研发阶段批准上市

首次获批国家/地区

加拿大

首次获批日期2022-01-21

1,621

项与遗传性肿瘤综合征相关的临床试验

NCT01199250 / Not yet recruitingN/AIIT

Specialized Program of Research Excellence (SPORE) in Endometrial Cancer

This research study is studying biomarkers in samples from patients with endometrial cancer. Studying samples from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.

开始日期2100-01-01

申办/合作机构

The Gynecologic Oncology Group

[+1]

NCT01445275 / Not yet recruitingN/AIIT

A Comparative Effectiveness Study of Cancer Risk Management for Women at Elevated Genetic Risk of Ovarian Cancer

This research trial collects information about types of treatment and the cost of these procedures in women with elevated genetic risk for ovarian cancer who participated on the Gynecology Oncology Group (GOG)-0199 trial. Gathering information about women at elevated genetic risk for ovarian cancer may help doctors learn more about risk reduction procedures and the cost of these procedures.

开始日期2100-01-01

申办/合作机构

The Gynecologic Oncology Group

[+1]

NCT06198296 / Not yet recruiting临床1期IIT

Immunotherapy For Adults With GPC3-Positive Solid Tumors Using Interleukin-15 And -21 Armored Glypican-3-Specific Chimeric Antigen Receptor Expressing Autologous T Cells

The body has different ways of fighting infection and disease. No single way seems perfect for fighting cancers. This research study combines two different ways of fighting cancer: antibodies and T cells.
Antibodies are types of proteins that protect the body from infectious diseases and possibly cancer. T cells, also called T lymphocytes, are special infection-fighting blood cells that can kill other cells, including cells infected with viruses and tumor cells. Both antibodies and T cells have been used to treat patients with cancers. They have shown promise but have not been strong enough to cure most patients.
In order to get them to kill cancers more effectively, in the laboratory, the study team inserted a new gene called a chimeric antigen receptor (CAR) into T cells that makes them recognize cancer cells and kill them. When inserted, this new CAR T cell can specifically recognize a protein found on solid tumors, called glypican-3 (GPC3). To make this GPC3-CAR more effective, the study team also added two genes called IL15 and IL21 that help CAR T cells grow better and stay in the blood longer so that they may kill tumors better. When the study team did this in the laboratory, they found that this mixture of GPC3-CAR,IL15 and IL21 killed tumor cells better when compared with CAR T cells that did not have IL15 plus IL21 in the laboratory. This study will use those cells, which are called 21.15.GPC3-CAR T cells, to treat patients with solid tumors that have GPC3 on their surface.
The study team also wanted to make sure that they could stop the 21.15.GPC3-CAR T cells from growing in the blood should there be any bad side effects. In order to do so, they inserted a gene called iCasp9 into the FAST-CAR T cells. This allows us the elimination of 21.15.GPC3-CAR T cells in the blood when the gene comes into contact with a medication called AP1903. The drug (AP1903) is an experimental drug that has been tested in humans with no bad side-effects. This drug will only be used to kill the T cells if necessary due to side effects .
The study team has treated patients with T cells that include GPC3. Patients have also been treated with IL-21 and with IL-15. Patients have not been treated with a combination of T cells that contain GPC3, IL-21 and IL-15. To summarize, this study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.
The 21.15.GPC3-CAR T cells are an investigational product not yet approved by the Food and Drug Administration.

开始日期2026-01-01

申办/合作机构

Baylor College of Medicine

[+1]

100 项与遗传性肿瘤综合征相关的临床结果

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100 项与遗传性肿瘤综合征相关的转化医学

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0 项与遗传性肿瘤综合征相关的专利（医药）

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132,011

项与遗传性肿瘤综合征相关的文献（医药）

2024-12-01·The Journal of dermatological treatment

Evaluation of psoriasis patients with long-term topical corticosteroids for their risk of developing adrenal insufficiency, Cushing's syndrome and osteoporosis.

Article

作者: Betul Erdem ; Muzeyyen Gonul ; Ilknur Ozturk Unsal ; Seyda Ozdemir Sahingoz

PURPOSE:

In this study, we will investigate the possible side effects of psoriasis patients using long-term topical corticosteroids (TCS) such as adrenal insufficiency, Cushing's Syndrome (CS) and osteoporosis and determine how these side effects develop.

MATERIAL AND METHODS:

Forty-nine patients were included in the study. The patients were divided into two groups based on the potency of the topical steroid they took and the patients' ACTH, cortisol and bone densitometer values were evaluated.

RESULTS:

There was no significant difference between the two groups regarding the development of surrenal insufficiency, CS and osteoporosis. One patient in group 1 and 4 patients in group 2 were evaluated as iatrogenic CS. ACTH stimulation tests of these patients in group 2 showed consistent results with adrenal insufficiency, while no adrenal insufficiency was detected in the patient in Group 1. Patients who used more than 50g of superpotent topical steroids per week compared to patients who used 50g of superpotent topical steroids per week. It was identified that patients who used more than 50g of superpotent topical steroids had significantly lower cortisol levels, with a negatively significant correlation between cortisol level and the amount of topical steroid use (p < .01).Osteoporosis was detected in 3 patients in group 1 and 8 patients in Group 2. Because of the low number of patients between two groups, statistical analysis could not be performed to determine the risk factors.

CONCLUSIONS:

Our study is the first study that we know of that investigated these three side effects. We have shown that the development of CS, adrenal insufficiency and osteoporosis in patients who use topical steroids for a long time depends on the weekly TCS dosage and the risk increases when it exceeds the threshold of 50 grams per week. therefore, our recommendation would be to avoid long-term use of superpotent steroids and to choose from the medium-potent group if it is to be used.

2024-12-01·Bioengineered

The cadmium tolerance and bioaccumulation mechanism of Tetratostichococcus sp. P1: insight from transcriptomics analysis.

Article

作者: Eri Sahabudin ; Shohei Kubo ; Muhamad Ali Muhammad Yuzir ; Nor'azizi Othman ; Fazrena Nadia Md Akhir ; Kengo Suzuki ; Kohei Yoneda ; Yoshiaki Maeda ; Iwane Suzuki ; Hirofumi Hara ; Koji Iwamoto

Cadmium (Cd) has become a severe issue in relatively low concentration and attracts expert attention due to its toxicity, accumulation, and biomagnification in living organisms. Cd does not have a biological role and causes serious health issues. Therefore, Cd pollutants should be reduced and removed from the environment. Microalgae have great potential for Cd absorption for waste treatment since they are more environmentally friendly than existing treatment methods and have strong metal sorption selectivity. This study evaluated the tolerance and ability of the microalga Tetratostichococcus sp. P1 to remove Cd ions under acidic conditions and reveal mechanisms based on transcriptomics analysis. The results showed that Tetratostichococcus sp. P1 had a high Cd tolerance that survived under the presence of Cd up to 100 µM, and IC₅₀, the half-maximal inhibitory concentration value, was 57.0 μM, calculated from the change in growth rate based on the chlorophyll content. Long-term Cd exposure affected the algal morphology and photosynthetic pigments of the alga. Tetratostichococcus sp. P1 removed Cd with a maximum uptake of 1.55 mg g^-1 dry weight. Transcriptomic analysis revealed the upregulation of the expression of genes related to metal binding, such as metallothionein. Group A, Group B transporters and glutathione, were also found upregulated. While the downregulation of the genes were related to photosynthesis, mitochondria electron transport, ABC-2 transporter, polysaccharide metabolic process, and cell division. This research is the first study on heavy metal bioremediation using Tetratostichococcus sp. P1 and provides a new potential microalga strain for heavy metal removal in wastewater.[Figure: see text]Abbreviations:BP: Biological process; bZIP: Basic Leucine Zipper; CC: Cellular component; ccc1: Ca (II)-sensitive cross complementary 1; Cd: Cadmium; CDF: Cation diffusion facilitator; Chl: Chlorophyll; CTR: Cu TRansporter families; DAGs: Directed acyclic graphs; DEGs: Differentially expressed genes; DVR: Divinyl chlorophyllide, an 8-vinyl-reductase; FPN: FerroportinN; FTIR: Fourier transform infrared; FTR: Fe TRansporter; GO: Gene Ontology; IC50: Growth half maximal inhibitory concentration; ICP: Inductively coupled plasma; MF: molecular function; NRAMPs: Natural resistance-associated aacrophage proteins; OD: Optical density; RPKM: Reads Per Kilobase of Exon Per Million Reads Mapped; VIT1: Vacuolar iron transporter 1 families; ZIPs: Zrt-, Irt-like proteins.

2024-12-01·Renal failure

Value of original and modified pathological scoring systems for prognostic prediction in paraffin-embedded donor kidney core biopsy.

Article

作者: Jinghong Tan ; Huanxi Zhang ; Longshan Liu ; Jun Li ; Qian Fu ; Yan Li ; Chenglin Wu ; Ronghai Deng ; Jiali Wang ; Bowen Xu ; Wenfang Chen ; Shicong Yang ; Changxi Wang

BACKGROUND:

No study has validated, compared and adapted scoring systems for prognosis prediction based on donor kidney core biopsy (CB), with less glomeruli than wedge biopsy.

METHODS:

A total of 185 donor kidney CB specimens were reviewed using seven scoring systems. The association between the total score, item scores, score-based grading, and allograft prognosis was investigated. In specimens with less than ten glomeruli (88/185, 47.6%), scoring systems were modified by adjusting weights of the item scores.

RESULTS:

The Maryland aggregate pathology index (MAPI) score-based grading and periglomerular fibrosis (PGF) associated with delayed graft function (DGF) (Grade: OR = 1.59, p < 0.001; PGF: OR = 1.06, p = 0.006). Total score, score-based grading and chronic lesion score in scoring systems associated with one-year and 3-year eGFR after transplantation. Total-score-based models had similar predictive capacities for eGFR in all scoring systems, except MAPI and Ugarte. Score of glomerulosclerosis (GS), interstitial fibrosis (IF), tubular atrophy (TA), and arteriolar hyalinosis (AH) had good eGFR predictive capacities. In specimens with less than ten glomeruli, modified scoring systems had better eGFR predictive capacities than original scoring systems.

CONCLUSIONS:

Scoring systems could predict allograft prognosis in paraffin-embedded CB with ten more glomeruli. A simple and pragmatic scoring system should include GS, IF, TA and AH, with weights assigned based on predictive capacity for prognosis. Replacing GS scores with tubulointerstitial scores could significantly improve the predictive capacity of eGFR. The conclusion should be further validated in frozen section.

1,159

项与遗传性肿瘤综合征相关的新闻（医药）

2024-04-03

·PRNewswire

INTERACT COALITION FORMED TO ADVANCE PATIENT ACCESS TO GENETIC TESTING FOR HEREDITARY CANCER RISK

SAN DIEGO, April 3, 2024 /PRNewswire/ -- Leading commercial organizations and patient advocacy groups in the field of cancer genetics today announced the founding of the Inter-Organization Cancer Genetics Clinical Evidence Coalition (INTERACT), a coalition whose mission is to increase evidence-based access to genetic testing for people with or at risk of hereditary cancers. Founding laboratory members include organizer Ambry Genetics, a subsidiary of REALM IDx, Illumina, Myriad Genetics, and Quest Diagnostics. Volpara Health has also recently joined the coalition. Founding patient advocacy organization members include AliveAndKickn and FORCE. The coalition seeks to provide a collective voice in support of the progression of medical professional and industry guidelines for genetic testing for inherited mutations that increase cancer risk. With growing insight into the role of genetic testing in cancer risk management and treatment, the population of individuals who benefit from knowing their genetic mutation status continues to increase. As leaders in the genetic testing and hereditary cancer field, the founding members believe it is their responsibility to help drive awareness and inform changes that will equalize access for those whose outcomes could benefit most from testing. One of the primary objectives of INTERACT is to ensure policy and guidelines keep pace with the growing body of evidence surrounding inherited cancer risk. Hereditary cancer genetic testing has been shown to improve outcomes by identifying those most at risk and informing management strategies. For instance, patients who test positive for a BRCA1 or BRCA2 mutation have up to 87% lifetime risk for breast cancer, and up to 40% lifetime risk for ovarian cancer.1,2 In addition, there are numerous other genes that increase risk for various forms of cancer. Armed with this information, patients and physicians can improve management through increased surveillance, chemoprevention, targeted therapeutics or risk-reducing surgical measures. As an example, studies have shown that prophylactic mastectomy in BRCA1/2 mutation carriers results in up to a 97% reduction in the risk for contralateral breast cancer, while salpingo-oophorectomy reduced ovarian cancer incidence by 69-100%.1,2 Despite the benefits of a patient and their provider knowing mutation status, disparities in access and uptake of cancer genetics services are well documented.3 INTERACT intends to improve access to genetic testing, with the goal of reaching vulnerable populations who may not currently be aware of their risk or their need for increased screening or other interventions. "With Lynch syndrome, one of the most common hereditary cancer syndromes, patients have up to 80% lifetime risk for colorectal cancer4, but an estimated 95% of at-risk individuals have not been identified5," said Robin Dubin, Executive Director of AliveAndKickn. "To really improve survival rates with informed screening strategies, we need to help drive education and policies that support genetic testing for all those at risk." Among the challenges to broadening access to genetic testing for hereditary cancer risk is a time lag in updating guidelines and medical policies after the publication of new medical literature. INTERACT will work to bring these differences to the attention of guideline committees and medical professional societies in an effort to bridge the gaps and reduce disparities in access to appropriate testing nationwide. About INTERACT The mission of INTERACT is to bring together specialized genetic testing laboratories and patient advocacy groups to support the progression and evolution of medical policy and industry guidelines for cancer genetic testing. Our members are recognized institutions in the field of cancer genetics. Current commercial members include Ambry Genetics, a subsidiary of REALM IDx, Illumina, Myriad Genetics, Quest Diagnostics, and Volpara Health. Advocacy members include AliveAndKickn and FORCE: Facing Our Risk of Cancer Empowered. We seek to develop the evidence base and rationale to inform changes in cancer-related genetic testing policies to expand patient access to evidence-based testing. For more information, visit: References: elson HD, et al Ann Intern Med 2014 Bayraktar S and Arun B. The Breast. 2017 Allford A, et al Eur J Hum Genet 2014 Lowry, Moonshot Blue Ribbon Panel 2016 Contact: [email protected] SOURCE INTERACT Coalition

临床研究

2024-04-01

·循因缉药

Lucid:我的市场600亿美元！Exact Sciences:那我来了哟嘿嘿！

环球视野，深度视角点击图片了解详情！点击这里在此发布！在开始之前，跟大家分享一个恐怖故事：一位股东由于没有星标我们这个小破号，他竟然半年没收到推送！那么，各位股东星标了么？点赞了么？转发了么？在看了么？谢谢！《你！不！要！过！来！啊！》这是曾经的标题，呃，我觉得太标题党了，各位股东觉得呢？看完再下结论。2024年3月28日，知名精准医学公司Exact Sciences宣布推动研发非内镜食管癌和癌前病变筛查技术。此时，一位名叫Lucid的靓仔晕了过去。有，且仅有大家其实并不了解，EAC（Esophageal Adenocarcinoma）食管腺癌也有早期癌前病变，且可以被早期发现。一般认为GERD（Gastroesophageal Reflux Disease，胃食管返流病）是一种相对常见的疾病，这种疾病会发展为Barrett’s Esophagus（Barrett食管炎）这种癌前病变。但是，很遗憾，早期阶段的食管腺癌并不会被发现。原因有二：第一，需要内窥镜+病理活检才能发现病变，早期很多患者很少会选择这种检查方式。毕竟大家谁闲着没事去往肚子里插根管子，再拽下块肉来，想想就难受。第二，缺乏合适的无创筛查方式。相对于乳腺癌的影像学筛查、宫颈癌的HPV检测以及结直肠癌五花八门的早筛产品，EAC早筛早诊产品很少。因此，美国人民的EAC发病率持续上升。相较于1975年，发病率上升了500%！那么，有没有人能够改变这一切呢？这您可问对人了！这不巧了么，咱们之前聊过的Lucid就是有办法解决这个痛点！Lucid开发了两款产品，EsoCheck和EsoGuard。EsoCheck是一款专用的采样器械，类似有尾巴胶囊。患者可以通过吞咽进入食管-胃交界处，然后由医生操作注射器将空气通过胶囊尾部的管子注入胶囊中。此时，胶囊充气，形成一个气球状采样器，然后拉出患者体内。食管壁上的细胞被刮下，随气囊一起装入样本管中寄回Lucid。此时，Lucid的中心实验室将使用一项EsoGuard LDT检测来对患者的样本进行检测。EsoGuard是基于NGS技术的甲基化检测，目标基因是VIM和CCNA1两个基因上的31个位点，使用的是Illumina MiSeq平台。能把EAC早筛/早诊采样器械（FDA批准）和检测方法结合的，阿美莉卡有，且仅有Lucid一家。当然，Lucid也有且仅有这一款产品。那么，Lucid做的怎么样呢？艰难求生2021年10月18日，Lucid完成上市，当时的股价是14美元/股。我们先来看看Lucid 给大家画的饼，也就是这个市场有多大。根据Lucid测算，按照3000万高危人群测算，美国的可触达市场规模（TAM）就有600亿之巨！然而，说起来你们可能不信，面对如此庞大的空白的市场Lucid 竟然没有爆卖...根据Lucid年报，2021/2022两年时间公司的营收都只能在300万左右徘徊。难啊...Lucid面对如此窘境，开始苦口婆心的招商。他告诉经销商们，开一个小诊所卖Lucid检测一周只要有1.7个检测你就回本了！终于，功夫不负有心人！2023年3月26日，Lucid Dx发布公告，全年的营收达到了245.2万美元。虽然不多，但是却是2022年的5.5倍！一瞬间，泪目了。巨头的“魔爪”Lucid Dx还没来得及开心两天，更让人泪目的事情发生了。对，没错，就是咱们开头提到的。Exact Sciences于2024年3月28日宣布，推动研发非内镜食管癌和癌前病变筛查技术。就你小子说的600亿美元TAM对吧？嘿嘿，我来啦~Exact Sciences采用了跟Lucid Dx类似的方法学，一样使用了专用的取样装置和一套甲基化检测方法。其中取样设备同样使用了一个可吞咽的胶囊装置，同样带有尾巴。不同的是，胶囊有一个可以降解的外皮+类海绵内球组成。吞咽后，经过消化液的作用，海绵球释放，然后通过食管取出，自然刮取内壁细胞。与Lucid Dx的EsoCheck相比省去了“打气”环节，省事又规避了专利。同时，通过352个样本的训练组找到了3个基因的甲基化位点作为最终候选检测标志物：NDRG4、VAV3和ZNF682。一番操作，在测试集中，拿到了敏感性88%，特异性84%的好成绩。呃，确实是不错了，虽然跟Lucid的结果略有差异。不过，以Exact打磨产品的能力，这一切都是第一步。更为关键的是，这货找到的合作人是Mayo Clinic。他真的，我哭死。对于Lucid 来说，目前最好的消息是，Exact的产品还在开发中。坏消息是，一旦商业化，面对Exact 庞大的商业化团队，Lucid几无胜算。所以，最开始的开始，我想的那个标题《你！不！要！过！来！啊！》是不是有点贴切了呢？最后我们最后还是说两句，一个研发型的公司在初期一定会尽量节省成本，以求开发出一个好的产品。研发阶段，可以保密。然而，一旦开始商业化，一切就由不得你了。这个阶段，拼的就是速度。拼抢占市场的速度、拼渗透市场的速度、拼产品迭代的速度。如果商业化速度不够快，别说开发新的领域了，新产品都迭代不出来。这时候，要是来了个大鳄，那就真的是降维打击了。好了，Lucid说的就是你，别哭了。看看有没有人收购吧，毕竟也没剩下多少市值了...最后，奉上Lucid合集。Lucid|说起来你可能不信，纳斯达克上市NGS诊断公司，Q3营收仅为7.6万美元乌龙记希望我这嘴没有开过光...END啊对对对对，扫描这货能联系到我近期文章：说好一起静悄悄，你却偷偷中了标一场秘密简报会，一起针对药明们的阳谋基因检测“样本外送”之殇煎熬的2023与煎熬的2024相关资料：注1：https://investor.exactsciences.com/investor-relations/press-releases/press-release-details/2024/Exact-Sciences-Advances-Development-of-Non-Endoscopic-Oncoguard-Esophagus-Test-to-Screen-for-Esophageal-Cancer-and-Pre-Cancer/default.aspx注2：https://ir.luciddx.com/2021-10-18-Lucid-Diagnostics-Announces-Closing-of-Nasdaq-Initial-Public-Offering注3：Lucid往期财报

2024-04-01

·PRNewswire

TSC Alliance® Announces 23rd Annual Step Forward to Cure TSC®

Events will raise funds for tuberous sclerosis complex (TSC) research and community support programs SILVER SPRING, Md., April 1, 2024 /PRNewswire/ -- As part of its 50th anniversary activities, the TSC Alliance® will present its 23rd Annual Step Forward to Cure TSC® event series, which will take place between April 13 and May 19. Nobelpharma is the 2024 Title Sponsor. Registrants can participate virtually from anywhere in the world or at 13 in-person locations across the United States. Proceeds will benefit the TSC Alliance's research efforts to find new treatments for tuberous sclerosis complex (TSC) and to offer an array of support programs and services. Tuberous sclerosis complex is a rare genetic disease that affects people at all stages of life. TSC causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin, eyes and lungs. Changes in the brain caused by TSC have the biggest impact on quality of life, from seizures and developmental delays to intellectual disabilities, behavioral challenges and autism. In fact, TSC is the leading genetic cause of epilepsy. A strong correlation also exists between TSC and autism—an estimated 40-50% of individuals with TSC have autism spectrum disorder. "Each year, our Step Forward to Cure TSC events offer easy opportunities to support our cause," explained Kari Luther Rosbeck, TSC Alliance President & CEO. "While the events raise much-needed funding, they also provide a platform for thousands of families, individuals and supporters to build camaraderie, learn from each other and educate their local communities about the disease." This year's in-person locations include Fishers, IN (April 13); Nashville, TN (April 27); Salt Lake City, UT (April 27); Renton, WA (April 28); Rock Hill, SC (May 4); Shepherdstown, WV (May 11); Northglenn, CO (May 11); Irwindale, CA (May 18); Chicago, IL (May 18); Tulsa, OK (May 18); West Chester, PA (May 18); Rockville, MD (May 19); and Carrollton, TX (May 19). "Bringing widespread awareness to a rare disease such as TSC requires a strong commitment to growing and sustaining a vibrant community of people who understand—or wish to learn about—the condition and who strive to meet the needs of those affected," said Yoshiki Kida, President & CEO of Nobelpharma America. "The TSC Alliance's Step Forward to Cure TSC campaign offers a fun and healthy way for anyone to have a positive impact on achieving that goal. Nobelpharma America is proud to support the TSC Alliance in this very important effort." To register online and learn more, visit StepFowardtoCureTSC.org. About the TSC Alliance The TSC Alliance is an internationally recognized nonprofit that improves quality of life for everyone affected by tuberous sclerosis complex by catalyzing new treatments, driving research toward a cure and expanding access to lifelong support. For more information, visit tscalliance.org. Contact Jaye Isham Chief Outreach Officer TSC Alliance (301) 562-9890 [email protected] SOURCE TSC Alliance