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CureDuchenneMD
United States
Приєднався 9 жов 2009
CureDuchenne is a national nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. Duchenne is a progressive muscle-wasting disease that impacts 1 in 3,500 boys. Boys are usually diagnosed before age 5, in a wheelchair by 12 and most don't survive their mid-20s. Currently, there are no approved treatments or cure for Duchenne. CureDuchenne funds promising research to find a cure for Duchenne. For more information, visit www.CureDuchenne.org
Navigating Duchenne treatment: AGAMREE & Catalyst Pathways
Join us for this pre-recorded presentation and Q&A discussing to learn about a new FDA-approved treatment for Duchenne muscular dystrophy. During the session, Catalyst Pharmaceuticals will provide an overview of how this novel corticosteroid works and its efficacy in Duchenne.
You will also learn about the journey to comprehensive support and access through the well-established Catalyst Pathways patient support program.
You will also learn about the journey to comprehensive support and access through the well-established Catalyst Pathways patient support program.
Переглядів: 108
Відео
Dyne Therapeutics DELIVER trial of DYNE-251 in Duchenne
Переглядів 205Місяць тому
Dyne Therapeutics shares with the community recent efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne.
ITF THERAPEUTICS| DUVYZAT Update
Переглядів 238Місяць тому
CureDuchenne Webinar | ITF THERAPEUTICS Patient Community Update: An overview of DUVYZAT June 26, 2024
The 2024 CureDuchenne FUTURES National Conference
Переглядів 217Місяць тому
The 2024 FUTURES National Conference brought together over 800 participants, both in-person and virtually, uniting the Duchenne muscular dystrophy community under the theme of "Bright Futures." This short video captures the essence of the event, showcasing heartfelt moments of connection, cutting-edge research presentations, and inspiring testimonials from families and experts. Attendees explor...
Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys
Переглядів 550Місяць тому
Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys
FDA Decision on Sarepta's ELEVIDYS Webinar
Переглядів 1,6 тис.Місяць тому
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. This therapy, while not a cure, aims to improve the quality of life for patients. The treatment involves a single dose, but patients must undergo extensive pre-treatment screening to help mitig...
Napa in Newport 2024
Переглядів 45Місяць тому
Experience the highlights of Napa in Newport 2024, held on April 20, at Pendry Hotel. This 10th-anniversary celebration brought together Napa Valley's finest wines and Newport Beach's coastal elegance to support CureDuchenne. Culinary Excellence: Chef Charles Phan of The Slanted Door delighted guests with Vietnamese-inspired dishes, perfectly paired with wines from Alpha Omega Winery. Wine Show...
2024 FUTURES New Therapeutic Approaches to Address Unmet Needs
Переглядів 1812 місяці тому
Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.
2024 FUTURES Preparing for the Unexpected, Responding to Emergencies
Переглядів 1232 місяці тому
A Duchenne emergency case study, the steps to take, and the signs to watch for. From implementing an emergency plan and following it to effective communication with the Emergency Room to get the care you need. A review of the cautions and a proactive plan of care from specialists.
2024 FUTURES Multidisciplinary Proactive Care in Duchenne
Переглядів 1302 місяці тому
Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.
2024 FUTURES Responding to AAV Based Treatment Challenges
Переглядів 2282 місяці тому
A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.
2024 FUTURES Exon skipping
Переглядів 2142 місяці тому
A brief introduction on the rationale behind exon skipping therapies, followed by an overview of current and investigative exon skipping therapies. Key insights were shared by Dr. Angel Angelov, Dr. Ash Dugar, Lianna Orlando, PhD, Dr. Kevin M. Flanigan, Laura Torrente, PhD, Alayna Tress, MPH, Karin Lucas, PhD, Mahasweta Girgenrath, Phd, Jessica Duis, MD, Husam Younis, PharmD, PhD.
2024 FUTURES GENE Therapy panel
Переглядів 3502 місяці тому
Gene Therapy Approaches to Treatment We covered the rationale behind gene therapy approaches for Duchenne and received updates from Pfizer, Sarepta Therapeutics, REGENXBIO Inc., and Solid BioSciences.
2024 FUTURES - Clinical Trial Design, Lessons Learned and Implications for the Future
Переглядів 642 місяці тому
An analysis of combined clinical trial data and what it can teach about clinical trial design. What has gone right and what should be changed in the future?
2024 FUTURES - Therapies to Preserve and Protect Muscle
Переглядів 2212 місяці тому
2024 FUTURES - Therapies to Preserve and Protect Muscle
2024 FUTURES - CureDuchenne LINK - What We Are Learning Through Your Participation
Переглядів 692 місяці тому
2024 FUTURES - CureDuchenne LINK - What We Are Learning Through Your Participation
Breakout Session: 2024 FUTURES Steroid Use
Переглядів 972 місяці тому
Breakout Session: 2024 FUTURES Steroid Use
2024 FUTURES Action is Therapy Getting Involved
Переглядів 622 місяці тому
2024 FUTURES Action is Therapy Getting Involved
Sarepta Therapeutics le da la bienvenida a 2024 FUTURES - Un Mensaje Aspecial
Переглядів 562 місяці тому
Sarepta Therapeutics le da la bienvenida a 2024 FUTURES - Un Mensaje Aspecial
Sarepta Therapeutics Welcomes You to 2024 FUTURES - A Special Message
Переглядів 1492 місяці тому
Sarepta Therapeutics Welcomes You to 2024 FUTURES - A Special Message
Rolling Through the Magic: Exploring Disneyland in a Power Wheelchair
Переглядів 2013 місяці тому
Rolling Through the Magic: Exploring Disneyland in a Power Wheelchair
CureDuchenne Webinar W/ italfarmaco | Community Update with ITF Therapeutics: Introducing DUVYZAT™
Переглядів 3704 місяці тому
CureDuchenne Webinar W/ italfarmaco | Community Update with ITF Therapeutics: Introducing DUVYZAT™
Ask Me Anything: Traveling with Duchenne with Marissa Penrod, Adam Malone and Alan Chaulet
Переглядів 1485 місяців тому
Ask Me Anything: Traveling with Duchenne with Marissa Penrod, Adam Malone and Alan Chaulet
CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients
Переглядів 1435 місяців тому
CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients
Pre-Recorded: Ask Me Anything: Fractures and Fatty Embolism Syndrome (FES) with Dr. Brenda Wong
Переглядів 3576 місяців тому
Pre-Recorded: Ask Me Anything: Fractures and Fatty Embolism Syndrome (FES) with Dr. Brenda Wong
Are you innovating new medicine to cure DMD or not
Innovate gene therapy to cure DMD as early as possible
The best moment during Futures!
I am proud of you!!
God Bless You!
God Bless You!!
Can Muscular Dystrophy treat?
My sun suffering from dmd. Please help me.l am from India.
Hi
What it is rarely about are the costs of these therapies. Would be nice if all these companies were as open about these very important matters too and explain why the costs are that high and how much profit they are going to make. We know what happened to Emflaza in the USA after a patent was bought. We know how Ireland refused to accept Translaran due to costs. Duchenne hits boys all over the world not covered by any insurrance. Nice to have medications that could help in the near future, but how will it help if there are no funds to obtain them. How are all these companies going to deal with that and what do they think it all will cost society. I think what Ireland did with Translarna is not only sort of brave but also good so to show society is not going ot accept rather absurd costs for very little real gain.
No, this is disgraceful and evil! Stop disguising eugenics. They're already aborting more black people than white, they already disallow disabled people to be born, more LGBTQIA are aborted than straight, more females are aborted than males, all advertisements of designer babies are images of white males. There's advertisements of anti aging which has nothing to do with sicknesses or diseases, if it was only about sicknesses and diseases they wouldn't of ever thought of touching the human germline. So don't bother denying this is racism, sexism, ableism, communism, eugenics and capitalism. They do not have the right to mess with a person's genes just because they perceive something about them as a "problem". Autism is not a problem, society claims it values all humans equally, since society wants to rub us out of existence you clearly do not see us as equal, these are full of discriminatory thoughts like Hitler doing eugenics. The problem is clearly their view of autism and other primarily genetic conditions. I love being autistic, I wouldn't give it up for the world. Molecular biologist Miroslav Radman writes, "Mutagenesis has traditionally been viewed as an unavoidable consequence of imperfections in the process of DNA replication and repair. But if diversity is essential to survival, and if mutagenesis is required to generate such diversity, perhaps mutagenesis has been positively selected for throughout evolution." Do you really want to bring us to extinction? Evelyn Fox Keller explains: "We now know that mechanisms for enduring genetic stability are a product of evolution. Yet a surprising number of mutations in which at least some of these mechanisms are disabled have been found in bacteria living under natural conditions. Why do these mutants persist? Is it possible that they provide some selective advantage to the population as a whole? Might the persistence of some mutator genes in a population enhance the adaptability of that population? Apparently so. New mathematical models of bacterial populations in variable environments confirm that, under such conditions, selection favors the fixation of some mutator alleles and furthermore, that their presence accelerates the pace of evolution." The mutants behind autism and other conditions like Down Syndrome offer some great advantages to the human race, diminishing the genes is a great risk because without those mechanisms there is no asurety of genetic stability pushing us in the direction of extinction, and delay the adaptation process. Psychologist Howard Gardner warns: "With the coming of age of genetics, the danger magnifies. Beyond doubt we will discover genes that are important for reading alphabetical scripts; and there is already evidence that a small set of genes may be related to reading problems. As with the brain evidence, such information can be helpful for early intervention; but it could easily be used for stigmatising purposes. Indeed, it might become relevant for marriage prospects, holding a job, securing insurance, or even eugenic purposes. And no doubt, especially in our interventionist society, individuals with a genetic predisposition for reading problems will look into different kinds of genetic engineering or therapy. It is possible that such interventions will work and have no negative side effects, but it is perhaps more likely that they will have unanticipated effects. And we might even want to consider which valued human abilities - eg. spatial or pattern recognition skills - might be placed at risk were we to target our interventions specifically at reading disorders." Do you really want to destroy all alternative perceptions and ways of thinking? Don't you know how many abilities you are going to destroy and how impoverished you are going to make our world because of your cultural myopia? Each time they have tried playing God they have only caused harm. Who caused the climate change? Scientists playing God trying to control nature, did these Gods anticipate the climate change? You are not only messing with humans, this whole earth is interconnected, you are messing with the entire ecosystem, with all life. How many species have been brought to extinction because of humans manipulating nature? there's endangered species today thanks to humans manipulating nature. If we fail to understand and take care of the natural world, it can cause a breakdown of these systems and come back to haunt us in ways we know little about. A critical example is a developing model of infectious disease that shows that most epidemics - AIDS, Ebola, West Nile, SARS, Lyme disease and hundreds more that have occurred over the last several decades - don’t just happen. They are a result of things people do to nature. The diseases you claim you want to cure were caused by doing this, so why are you doing it again? Was the world ready for COVID-19 to strike? I doubt it. World War II was caused by eugenics, why are you following Adolf Hitler's steps? Mutations are not random or accidental, malaria is endemic in Africa and Africans have developed mutations that protect them from malaria through adaptation, the sickle cell mutation is a defence against malaria, Europeans don't have these mutations, if a European goes to Africa they are more likely to get a disease, what would of happened to Africans if you eliminated that defence mechanism? The Africans would of been eliminated. It was mutations that enabled the Europeans to survive the 14th century bubonic plague. Editing one gene may cure a disease but at the same time make them more susceptible to other diseases. Do you really believe mutations that enable survival are really just accidental and random as these assume? Again, this is wicked and pure evil to think we don't deserve to be born just because we are different. CRISPR-Cas9 is a direct violation of human rights, especially human autonomy. They are full of discriminatory thoughts and don't you touch us without our consent!
Very good, but what I am missing in all these things are answers about the future. Nothing definitive but I guess most parents would like to know when thngs could potentially come to market if Phase III studies work out fine.
Mike needs a better Mic.
All this amounts to a couple of extra years where kids, treated in time, can walk. So this disease even with givinostat is devastating. But we are getting somewhere out of the predisone/prednisone era where side effects are so bad you can wonder if you want to give your kid these medications. I wonder where Vamorolone+Givinostat will get us. Or with upcoming treatments that are in phase three, like pamrevlumab that focusses on fibrosis and other treatments. Like some that do take over the function of Dystrophin. May be the combination of these will get people really on the way.
I am the mother of a 14 years old boy with DMD who is no longer ambulant for years and I am very eagerly waiting for the Vamorolone/Givinostat combo, hoping it could hopefully limit the damage caused by the disease.
If they cure it they'll be out of a job
I'm 40 years old and have tried my entire life to get on a trial study you always be too well too sick too old to Young
I have 2sons who are suffering from this monster but please God make away
Where is the link for registration? Please advise
Finding therapies is one part of the story making these affordable for patients is another . What are Pharma companies doing to address the patient access
Please get cure
i have dmd 34 years old
Hi dear. What do u use? My nephew has it
@@komugishabrillian5616 nothing just prednisone
@@Brisingr how many times do you take in a day
@@komugishabrillian5616 once a day 5mg then 10 the next day then 5...
@@Brisingr Thank you so much. Many More Blessings
Please please do something. My son suffering from dmd.he is 11+ and he walked her self now. Please please do something
Please visit India also ..
Please do something for curing DMD
Please do something fast for curing DMD please
【p】【r】【o】【m】【o】【s】【m】
how you doig?~nice !take care. buddy! :))
My 4.5 year old cousin is suffering from DMD, please help. We are from India. Is it possible to get the treatment in India?
We need a cure for the boys
www.cureduchenne.org/getzlaf-golf/
Thank you for the Updates
Hi my son suffering from this DMD , is any cure for thi pls help why our scientists not find any cure for this if they find cure for Corona as early pls help pls find any treatment for this big problem many people suffering from this and his family can't do any thing
My brother has the duchenne too,please, if you scientists care about us,please help...
There putting a golf tournament come out have so fun. May 2 2022 it all nonprofit also they have wheelchair soccer team
Excuse me. 1. AMONDYS 45 2. EXONDYS 51 3. TRANSLARNA 4. VILTEPSO 5. VYONDYS 53 Is this medicine easy to get in the US? How about getting it from countries other than the United States? Is the price of medicine the same in all countries?
Beautiful presentation! My 10 year old grandson has DMD. Thank you for sharing!
Pls give supplements list
God bless them all.. Praying for a cure!
Is there any cure for them now? plz answer me
Contact doctor Akhigbe for your cure with his herbal medicine
Email him now
Can serial casting help for dmd patient
Contact doctor Akhigbe for your cure with his herbal medicine
Email him now
Free James
Hi, i think your looking for hank meijer!
Please find a cure.
When can we buy DMD drugs from the pharmacy
We got good improvement in muscular dystrophy patients cases for further information please contact me on WhatsApp 91 9322303271
My older brother died at fourteen cause of duchenne and my little brother has the same kind of duchene as my older brother
Please contact me on whatsapp 91 9322303271 I like to inform you that we got good improvement in SMA DMD LGMD patient cases with the help of acupressure massage therapy healing process power meditation and ayurvedic medicines for further information please contact me on
Don’t fall for it it’s a scam you cannot reverse The progression of The disease with acupuncture it’s just not possible
@@kantilalbhanushali7572 Stop scamming people
@@theinventor7027 🙏 you miss understood me. Please if you know any muscular dystrophy patients just ask them to connect me I will teach them how to do power meditation within a month they will find relief in pain and improve in movement my whatsapp 91 9322 30327 1
ua-cam.com/video/JuTZNx4VYGE/v-deo.html
Traditional healer who cured me from Muscular Dystrophy Disease, this is a testimony. you can also add him on Whatsapp. I was suffering from Muscular Dystrophy Disease for years. i suffered till i meet Dr EHI ORIRI the great. +233 20 280 0522 or Email him on drehioriri@gmail.com.
One month age doctors ensure my brother have Muscular Dustropy 😢😢😢😢😢 we are really shocked My family is just break down.my brother is only son in my family.... I don't just accept it... Yesterday my parents went to India for better treatment for my brother... please pay for him. I am from Bangladesh. years years
Where is gene therapy available How expensive it is
Yes I would love to know too, PLEASE let me know if you have any updates 😩🙏
I need help 😔 I have same problem
Please research Dr. Robert Morse on UA-cam🍇💕I wish you luck on your healing journey!
Traditional healer who cured me from Muscular Dystrophy Disease, this is a testimony. you can also add him on Whatsapp. I was suffering from Muscular Dystrophy Disease for years. i suffered till i meet Dr EHI ORIRI the great. +233 20 280 0522 or Email him on drehioriri@gmail.com.
Please contact me on WhatsApp 91 9322303271
@@kantilalbhanushali7572 sir aap har jagah kese😂😂
@@richardmilton3431 please help me reach out to him because me and my son are patients too 🇺🇬🇺🇬
Pls How can I get this medicine for my son need to contact you if possible
@Happiness Mtui If you're not already a member, please join the DuchenneXchange.org and check out the trial wizard to find a clinical trial that may be right for your son
Gluten causes every disease
Traditional healer who cured me from Muscular Dystrophy Disease, this is a testimony. you can also add him on Whatsapp. I was suffering from Muscular Dystrophy Disease for years. i suffered till i meet Dr EHI ORIRI the great. +233 20 280 0522 or Email him on drehioriri@gmail.com.
.Hi, happy day, please, I am with me, a child with Doshan disease.Please help me.I did a lot of analyzes and with me the analysis.Please help me.I want your phone number, email and WhatsApp number to contact you.I am tired a lot.Please stand by me.