ACTA2 Alliance’s Post

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

September has arrived with two crucial campaigns that need greater visibility within society. Brazil Potash, through its subsidiary Autazes Potassio Project, is fulfilling its role as a Corporate Citizen by promoting Green September and Gold September, reinforcing information that can save lives. Green September encourages organ donation, aiming to raise public awareness about the importance of this life-saving act. The campaign seeks to strengthen discussions on this topic and spread the information as widely as possible. In Brazil, organ donation is only performed after family authorization, making it essential to discuss your wish to be a donor with your family. Gold September, on the other hand, is a national initiative to raise awareness among parents, educators, and healthcare professionals about childhood cancer. According to data from the National Cancer Institute (Inca), cancer is already the leading cause of disease-related death (8% of the total) among children and adolescents aged 1 to 19 in Brazil. Early diagnosis is crucial for successful treatment. #PotassioDoBrasil #BrazilPotash #PotassioFertilizers #ESG #Potassio #PotassiumChloride #Amazonas #Autazes #AutazesPotashProject #LicensedForInstallation #GreenSeptember #GoldSeptember

Please wish beautiful sweet Iris and rare young lady a wonderful 16th birthday and help us spread awareness for Rare Disease Day! Double your donation up to $16K through Rare Disease Day, 2/29! curegm1.org/donate (option for no fees with PayPal Giving Fund) or https://lnkd.in/gktBZ5Vb Your help is critically important to all those dying due to GM1 gangliosidosis. You can help make a difference. Watch on YouTube: https://lnkd.in/gzYXfVij 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. Tragically, only one trial is still running, the remaining trial is in Phase 3, the final stage. To make matters worse, rare disease programs have been shuttered due to a downturn in biotech and gene therapy. We try to remain hopeful, but the reality is that this fight continues and more help is needed. Children and babies are dying while programs are shelved. The science is known on how to help and yet programs are stopped due to money. There are still many ways that we can contribute to help, including next generation therapies, newborn screening, continued advocacy, and more. Thank you. Please kindly share and spread the word. Music by BenSound. #curegm1 #raredisease #advocacy #birthdays #donate #give #hope #fightforlife #smallactionsbigimpact

LAST CHANCE to double your gift! Double your donation up to $16K through Rare Disease Day, 2/29! curegm1.org/donate (option for no fees with PayPal Giving Fund) or https://lnkd.in/gX6658UM Your help is critically important to all those dying due to GM1 gangliosidosis. You can help make a difference. 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. Tragically, only one trial is still running, the remaining trial is in Phase 3, the final stage. To make matters worse, rare disease programs have been shuttered due to a downturn in biotech and gene therapy. We try to remain hopeful, but the reality is that this fight continues and more help is needed. Children and babies are dying while programs are shelved. The science is known on how to help and yet programs are stopped due to money. There are still many ways that we can contribute to help, including next generation therapies, newborn screening, continued advocacy, and more. Thank you. Please kindly share and spread the word. #curegm1 #raredisease #advocacy #birthdays #donate #give #hope #fightforlife #smallactionsbigimpact

Please help wish a beautiful sweet and rare young lady a wonderful sweet 16! Double your donation up to $16K through Rare Disease Day, 2/29! curegm1.org/donate (option for no fees with PayPal Giving Fund) or https://lnkd.in/gX6658UM Your help is critically important to all those dying due to GM1 gangliosidosis. You can help make a difference. 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. Tragically, only one trial is still running, the remaining trial is in Phase 3, the final stage. To make matters worse, rare disease programs have been shuttered due to a downturn in biotech and gene therapy. We try to remain hopeful, but the reality is that this fight continues and more help is needed. Children and babies are dying while programs are shelved. The science is known on how to help and yet programs are stopped due to money. There are still many ways that we can contribute to help, including next generation therapies, newborn screening, continued advocacy, and more. Thank you. Please kindly share and spread the word. #curegm1 #raredisease #advocacy #birthdays #donate #give #hope #fightforlife #smallactionsbigimpact Music by BenSound

TeacherActive are raising money and awareness for the Cystic Fibrosis Trust. Cystic fibrosis (CF) is a chronic and progressive genetic disorder that primarily affects the respiratory and digestive systems. This condition results from mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene, leading to the production of thick and sticky mucus. This mucus can clog airways and ducts, causing various complications and affecting the overall health of individuals with CF. 🍋 Donate Today: Your financial support will directly contribute to ongoing research initiatives, medical advancements, and support programs for individuals and families affected by cystic fibrosis. 🌻Spread the Word: Share this post with your friends, family, and social networks. Awareness is a powerful tool, and together, we can reach more people, garnering increased support for this important cause. 💛 Donate Now and Be a Beacon of Hope! https://lnkd.in/ekv5iv_t Your generosity will not only fund critical research but also provide hope and support to those who need it most. Let's stand together, united in our mission to breathe hope into the lives of individuals with cystic fibrosis. #pridepassionpeople #TeacherActive #cysticfibrosis

Cockayne syndrome – type B (CSB) is a profoundly devastating and deadly genetic disorder that is tragically destroying the lives of children, stripping away the promise of their futures. Currently there is no cure or treatment for this rare disease and the only hope for children is development of gene therapy. In Viljem Julijan Association we are on a courageous mission to develop gene therapy for Cockayne syndrome – type B (CSB) and we have partnered with scientists from USA and Portugal who are dedicated to the creation of this vital treatment and are actively pursuing it. Our mission was inspired and initiated by a brave 5-years old girl named Karolina from Slovenia (Europe), who became the beacon of hope for all children with CSB around the world. To bring this vision into reality, the development of gene therapy necessitates substantial funding, amounting to €2,000,000 and in Viljem Julijan Association we have already raised €1,310,000 through the generosity of our supporters. Yet, the path ahead requires further financial assistance to continue this critical research and development. Please help us support the research and development of gene therapy for Cockayne syndrome – type B and contribute a donation. Contributions can be made through PayPal or the GoGetFunding platform (https://lnkd.in/dCH3PVax). By contributing a donation, you are not just offering financial aid; you are investing in hope, in life, and in the future of children affected by Cockayne syndrome – type B. Your generosity could be the key to unlocking a new horizon for many, offering them a chance at a brighter tomorrow. Dr Clévio Nóbrega Dr Christina Pacak #ViljemJulijanAssociation #GeneTherapy #CureForCSB #Treatment #Cockaynesyndrome #Karolina #Slovenia

Could we be your next charity partner? Or your next Charity of the Year? Do you share our vision? We envision a world where those living with NF2 will have access to cutting-edge solutions to live full and productive lives, free from daily physical and emotional pain or suffering. But, like many charities, we rely on donations to fund our research, We’re a very small charity and we are ENTIRELY volunteers, who are patients, family, friends, or carers of someone living with NF2. Everyone has lived experience of NF2. Research is key to developing new treatments and to finding a cure for those affected by NF2. With your help we can fund vital research and change the lives of adults and children affected by NF2 We are focused on promoting, facilitating, and financing gene therapy, cell therapy & immunotherapy research to develop new treatments or a cure to NF2. Our dedication to research and innovative treatments brings hope to patients and families We need to ensure the physical and emotional wellbeing of children & adults affected by NF2 without the need for invasive surgeries or treatments that may rob them of their nerve functions & senses one at a time. By supporting our innovative research through corporate fundraising, you might consider - • Nominate us as Charity of the year • Events – You could hold an event to benefit us, (eg Golf Days, charity evenings, raffles etc) • Support someone who works with you who has NF2 by donating or holding a fundraiser in their honour. • Corporate Donations – companies benefit from this also with their tax returns • Staff Fundraising – get staff involved in bake sales, coffee mornings or festive/themed sponsored fun days (think Christmas Jumpers!) – lots of ideas to raise funds! Let us know if you can help us in any way, or if you could nominate us to your employer or indeed if you run a business yourself, Or do you want to support someone who works with you who has NF2? We would LOVE to talk to you about how you can help us change the lives of those with NF2 related Schwannomatosis. Please message or email us clare@nf2biosolutions.org or joanne@nf2biosolutions.org #endNF2 #NF2awareness #neurofibromatosistype2 #schwannomatosis #NF2support #nf2 #nf2family #nf2warrior #LetstalkNF2 #nf2charity #nf2cure #nf2treatment #nf2gene #nf2chromosome22 #nf2trials #nf2diagnosis #nf2lifeexpectancy #nf2prognosis #nf2research #nf2ismyteam #nf2awarenessday #nf2journey #StrongerTogetherAgainstNF2

Help us make a lifesaving impact! The World Marrow Donor Association (WMDA) community volunteer on 50 vital projects geared towards enhancing the chances of patients finding their life-saving match donor and reducing the time it takes for a patient to receive a transplant. Each year, our efforts culminate in over 20,000 stem cell transplants, offering hope and second chances to countless individuals worldwide. However, with only 57 countries worldwide being able to access WMDA's knowledge and support, we believe there's much more we can do to save even more lives. Your support can turn possibilities into realities. By donating to us through GlobalGiving, you can directly contribute to our vision of access to life-saving cellular therapies for all patients. Every donation, no matter the size, has the power to transform lives and bring hope. Our focus for this Little by Little campaign is bringing our community’s knowledge to Africa. In a continent of 54 countries, only 2 have access to WMDA's knowledge and support. In 76 days, our community will be in Cape Town. We want to support as many hematologists, oncologists, cancer charities and sickle cell disease foundations in Africa in attending the conference and beyond. All donations between USD 5 and up to USD 50 will be matched by GlobalGiving at 50% until matching funds run out, or on April 12th at 11:59 pm EDT, whichever comes first. The Little by Little campaign has officially begun so don’t hesitate to donate! https://lnkd.in/e5zbgMk9 Please share so that we can reach more people and increase the chances for all ❤️ #globalgiving #charitablegiving #nonprofitfundraising #socialimpact #accesstohealthcare #makeadifference #bethechange #sharingiscaring

Absolutely, having healthy children is a blessing, and for those facing challenges due to conditions like GM1 gangliosidosis, support from individuals and organizations can make a significant difference. GM1 gangliosidosis is a rare genetic disorder that affects the nervous system and typically manifests in infancy or early childhood. The Cure GM1 Foundation is dedicated to funding research, raising awareness, and supporting families affected by GM1 gangliosidosis. Their efforts contribute to advancing scientific understanding of the condition and developing potential treatments or cures. Supporting organizations like the Cure GM1 Foundation can be done in several ways: Donations: Contributing financially to the foundation enables them to fund research projects, provide support services to families, and raise awareness about GM1 gangliosidosis. Volunteering: Offering your time and skills to help organize events, raise awareness, or assist with administrative tasks can be invaluable to organizations like the Cure GM1 Foundation. Advocacy: Advocating for increased research funding, promoting awareness about GM1 gangliosidosis, and supporting policies that benefit individuals and families affected by rare diseases can make a meaningful impact. Fundraising: Organizing fundraising events or campaigns within your community or workplace to support the Cure GM1 Foundation can help raise vital funds for their initiatives. Spreading Awareness: Sharing information about GM1 gangliosidosis and the work of the Cure GM1 Foundation through social media, community events, or educational seminars can help raise awareness and support for their cause. Every contribution, no matter how small, can make a difference in the lives of those affected by GM1 gangliosidosis and their families. If you're able to find it in your heart to support the Cure GM1 Foundation, you'll be helping to make a positive impact on the lives of individuals facing this rare genetic disorders. Click on Doug's post below to see the journey of Iris his daughter who has GM1 gangliosidosis. I have known Doug for 12 of the 16 years of his daughters Iris's life.