CureGRIN Foundation’s Post

LET'S CURE CUTE KIDS Fundraising for kids with genetic disease Do you know it's a privilege to be able to talk, see and walk because 1 in 20 Aussie kids suffer from genetic disorders such as cancer, neurological disorders or physical disorders? They don't have such a privilege and are dying young in pain hopelessly. Who am I and why am I raising fund? I am a new researcher at Children Medical Research Institute (CMRI) which aims to find cures for kids globally with genetic disease that has very limited treatment options or not at all. I am madly passionate about this research and fundraising activities. Because government funding is crazily competitive, our institute is completely run by the community and 82% of the money we receive goes directly to research. What do we do? Curious about the mechanism of disease to be able to treat its root cause, we mainly do basic science varying from stem cells, cell biology, and cell signalling to neurobiology, and embryology. We also translate our findings to the bedside with innovative research such as viral vectors and gene therapy. Our world-class international cancer research project (ProCan) aims to build the largest "public" library of cancer, to help choose the most effective treatment on the very first days for any child or adult with any type of cancer. In the next 2 decades, we expand our research not only genetic disease but also to infectious disease, kidney disease and metabolic disorders. - Learn more about our research teams at: https://lnkd.in/g4-QKq8Q - Learn more about real kids whom we help: https://lnkd.in/gVWqxmZa Interested in that vision? Do you want to give us another hand? Just by:  - Donating on my website below: As small as your one dollar can make a big impact. - Sharing my post or website with your friends and family. A sincere thank you for your generosity! Together we can make the impossible possible :) Thi Thi Anh Nguyen (MSc, B.Pharm) | PhD student  Sydney Pharmacy School  Faculty of Medicine and Health The University of Sydney | Sydney | NSW | 2006 Pharmacy Neighbourhood (K-block, Level 5) Westmead Hospital | Westmead | NSW | 2145 E thi.a.nguyen@sydney.edu.au ORCID ID 0000-0002-8508-8990

Did you know that a rare disease is defined as one that affects fewer than 200,000 people? Today, on Rare Disease Day, as we do every day, we spotlight NF2, a condition that impacts a small but resilient community of individuals worldwide. NF2 may be rare, but its impact is profound. It's a genetic disorder characterized by the growth of tumors on the nervous system, leading to hearing loss, balance issues, and sometimes even paralysis. Living with NF2 means facing daily challenges that most people can't imagine. But here's the thing: even though NF2 is rare, our hope for a cure is anything but. With your support, we're pushing forward the research in gene therapy at Nationwide Children's Hospital's Meyer Lab. Our mission is to change the lives of those battling NF2, one step closer to clinical trials and, ultimately, a cure. Join us in raising awareness and funds for NF2 research. Every donation, no matter how small, brings us closer to our goal. Let's show the world the power of unity and compassion in the face of rare diseases. Together, we can make a difference! Donate here: https://buff.ly/4c35L9w #RareDiseaseDay #NF2 #NF2Awareness #NeverForget2LivewithHope

A genetic health risk could mean you have a change in your DNA that makes you more likely to develop certain types of cancer, heart disease, neurological disorders, and other common health risks. Genetic testing to identify these risks is life-saving because knowing early can lead to earlier or more frequent screenings. It can even let you know if certain family members may also be at risk of developing these conditions. When you make a donation to HudsonAlpha's Annual Fund, you're supporting programs like the Smith Family Clinic for Genomic Medicine, that offer genetic testing services to help access this important health information. Make a donation by July 31 and your gift will be matched up to $10,000 by Freedom Real Estate and Capital: https://lnkd.in/gQugiifP #genetics #health #risk #annualfund

Double your donation up to $16K through Rare Disease Day, 2/29! curegm1.org/donate (option for no fees with PayPal Giving Fund) Your help is critically important to all those dying due to GM1 gangliosidosis. 10+ years ago, Iris was just about to start kindergarten when our world was completely changed forever and shaken by a fatal diagnosis of GM1 gangliosidosis. Doctors who were uninformed about juvenile GM1 told us that she had over 5-10 years left to live and she's still here, still fighting for every single day and moment. We founded the Cure GM1 Foundation in her honor and in honor of all those suffering from GM1 gangliosidosis. Significant strides have been made. There have been 4 clinical trials since 2019 and there were no trials prior to the creation of Cure GM1. Although only one trial is still running, the remaining trial is in Phase 3, the final stage. Many rare disease programs have been shuttered due to a downturn in biotech and gene therapy. We remain hopeful, but the reality is that this fight continues to evolve. There are still many ways that we can contribute to help, including next generation therapies, newborn screening, continued advocacy, and more. You can help us make a difference! #curegm1 #raredisease #advocacy #birthdays #donate #give #hope #fightforlife #smallactionsbigimpact

We NEED your SUPPORT to reach our goal of $200,000 by the end of 2023! On Friday, December 1st, the Cri du Chat Research Foundation (CDCRF) will celebrate its Annual Lucky Liam Gala at the Dyker Beach Clubhouse in Brooklyn, NY. The event is held in honor of Liam Leston, age 10, who lives with Cri du Chat syndrome (CdCS, also referred to as 5p Minus Syndrome), along with the many children and adults impacted by this rare disorder. Cri du Chat Syndrome is a severe rare neuro-developmental syndrome that is caused by a deletion of genes on the 5th chromosome. It impacts 1 in 15,000 newborns annually. The Syndrome causes many challenges, including muscle weakness and coordination deficits, apraxia of speech, swallowing issues and autism. Some children have major organ involvement such as heart and kidney disease, or suffer from brain seizures. The diagnosis of CdCS / 5p Minus is a spectrum disorder due to the wide range of symptoms, but all individuals require lifelong support even with today's interventions and therapies. The Cri du Chat Research Foundation is a 501c3 Non-Profit organization committed to developing a medical treatment. We are working towards a Gene therapy (replacing or repairing the function of a targeted gene or genes by introducing specific genetic material) and an RNA therapy (a new class of medication, like the coronavirus vaccines) which would create an impact at the genetic level for the CdCS Individual to minimize or cure symptoms of Cri du Chat Syndrome. Our goal of $200,000 by the end of 2023 will directly fund testing our therapeutic approaches in human derived stem cells and animal models... progressing another step closer to clinical trials with the end goal of helping those impacted by Cri du Chat / 5p Minus Syndrome. We are making incredible strides towards our goal, but will truly need "all hands on deck" to reach it... So Like, Donate, Share, Join us for an amazing event. And thank you everyone for your continued support!!! Your support will play a tremendous role in making this possible! Donate: https://lnkd.in/ewMe6ttz Tickets/Sponsor: https://lnkd.in/eMTgqd_k

Ten-day challenge to raise $10,000 to benefit sickle cell patients (Day 2) Thank you to my network who donated yesterday to help me push towards my $25,000 June 1st fundraising goal. There were 5 donations, including one from a repeat donor! Can we get to 10 donations today? Sickle cell disease is a group of inherited red blood cell disorders that affect hemoglobin, the protein that carries oxygen through the body. Normally, red blood cells are disc-shaped and flexible enough to move easily through the blood vessels. In sickle cell disease, red blood cells become crescent- or “sickle”-shaped due to a genetic mutation. These sickled red blood cells do not bend or move easily and can block blood flow to the rest of the body. The blocked blood flow through the body can lead to serious problems, including stroke, eye problems, infections, and episodes of pain called pain crises. (https://lnkd.in/dXNdf3Eq) Over 100,000 people affected in the US. Over 6 million people affected worldwide. Let’s help these patients. Donate here: https://lnkd.in/ev3CZxfj

Ten-day challenge to raise $10,000 to benefit sickle cell patients (Day 1) Over the past two months, I have sent 500+ (and counting) individual messages to my network to solicit support for my Timmerman Traverse for Sickle Forward campaign, in which we will be climbing Mt Kilimanjaro in the fall to benefit and raise awareness for sickle cell patients.  I am extremely grateful to everyone who has helped me raise $15,000 thus far! I would like to challenge my network to help me raise another $10,000 over the next ten days to help me reach my $25,000 June 1st midpoint goal. Breaking it down, just ten $100 (give or take) donations a day over ten days is one way we can achieve this goal. I also want to use this challenge to raise awareness for sickle cell disease each day. I would like to start with a statistic that keeps circulating back into my mind: 50-80% of untreated children in Africa will die before their 5th birthday. The first step in treating this disease is diagnosing the affected newborn. Please consider a donation using my fundraising link below. Proceeds will go to Sickle Forward who will facilitate diagnosis and early treatment of newborns in Africa.  $1 dollar = 1 infant screened. https://lnkd.in/ev3CZxfj

This holiday season, please consider helping my 18 month old son Max find a cure for the fatal Alexander Disease. Treatments are within reach and every dollar makes a huge difference towards bringing them to the children and families who are suffering. Thanks to donations by many of you, we are 51% towards our ambitious goal. With your support and the power of the community here, we hope to be able to fund the remaining experiments. Max was diagnosed with Alexander Disease at just 7 months old, after a 30 minute long seizure. This rare and fatal neurological disorder, a spontaneous mutation affecting 1 in a million, affects the brain and spinal cord, leading to symptoms like seizures, balance and motor issues, and developmental delays. Doctors gave Max 5 years. But we are not giving up, and have connected to scientists and families around the world who are making a difference. There is currently no cure for Alexander Disease, but there is hope. Promising new treatments are emerging, some of which proved effective in other neurodegenerative diseases, but need to be tested in AxD models. Crucial experiments are only held back by a lack of funds. So Max’ amazing mom Stephanie Holzhuber and I, together with other families, are raising money to support research into life-changing treatments, including at the Alexander Disease Lab at the University of Wisconsin, Harvard Medical School, UMass Chan, Western Sydney University and others. Every donation, big or small, brings us closer to a future for Max and all children with Alexander Disease. One where they can walk and talk and live. Please visit our GoFundMe page to learn more and, if you can, please donate or share: https://lnkd.in/gd62n6Cg #AlexanderDisease #EndAxD #HopeForMax #RareDisease #Giving #Fundraising #charity

Become a hero for Max. 7 months into his life, Max was diagnosed with the fatal Alexander disease. Since then, his parents Stephanie Holzhuber and Thomas Wagner have been moving mountains in their journey to find a cure. You can join them and help by : - bringing awareness about the research and studies about the disease - sharing the below post - donating to the fundraiser - connecting with Thomas Wagner and/or Stephanie Holzhuber Let's do it together, not only for Max, but all the kids and families impacted by the disease. https://lnkd.in/gkdTNAR5

Please consider DKMS on Giving Tuesday tomorrow: Clementine's parents' world shattered when, at just 8 months old, she received a life-threatening diagnosis of a rare genetic mutation, making her the first documented female with this condition. The future her parents envisioned for their daughter took a dark turn. Fortunately, a generous stem cell donor gave Clementine the future her parents wanted for her. Through donors like you, we were able to register 600,601 new potential lifesavers in 2023. Globally, 7,373 people were able to donate their blood stem cells to patients through DKMS to give recipients like Clementine a second chance at life. It costs nothing to register, but it costs DKMS $45 to process each registration. This Giving Tuesday, we invite you to support DKMS in expanding the donor registry with a financial contribution, providing other patients like Clementine, a life where their future is just as bright. https://lnkd.in/gx6TCMNg #givingtuesday #givingtuesdayclt #deletebloodcancer #impact #savealife