I have LVNC. Along w some neuromuscular thing which causes generalized muscle pain and weakness. On Fab Four drugs and hoping ef will come up from 25%. Thank
I love what he said about helping the doctors to help them. I sure hope there will be more advancements in medical research to not only treat symptoms but also to find a cure for this, and other, fatal conditions. I have a lot of faith in gene therapy for this and other conditions. It just makes sense.
So sad to see that this beautiful child has passed away. He was very visibly struggling in this clip. His lips appear blue. I hope and pray for a cure for this, and other, fatal illnesses before more precious kids like this lose their lives!
If I found out when I was only 6 weeks then tbh, I'd have an abortion. I wouldn't want to take the risk. Especially if I already had several healthy kids like these two.
This was an incredible interview, Injust wish it was longer and wish we learned more about their family and was baby C okay? I can only imagine what they've gone through. I hope they are well and have looked into stem cell therapy!! 💓💗❤️
Ears never stop growing in all humans. The ears aren't enlarged though, the rest of them is just small because the mitochondria in the cell doesn't work properly and it affects every muscle in the body, especially the biggest muscle the heart, which is usually how they die. Because their hearts are so small and every cell in the body is so exhausted. Like a random arythmia for example that could come out of nowhere can kill them. This lowers their life expectancy by a lot.
Thank you so much Abe and family for sharing your story. Here's to hoping for a favorable decision by the FDA regarding the use of elamipretide to treat Barth syndrome.
Thank you Abe and your parents for this great video that advocates for all Barth boys and their families. You did a great job! Pray for positive decision from FDA
For 6 years I have been battling with cardiovascular disease which almost took my life, but when I came across Dr. Gbenga, I decided to give a try to his herbal mixture, which I used for sometime but today am completely cured and strong, thank you Dr. Gbenga for your help.
Abe's story of the story of hundred with Barth Syndrome and 30 million with one of the other 7,000 Rare Diseases. Yea - what about the "risk " of a great outcome - let's seize it! #FDA #RareDisease #MLD #FMLD
That's a great story. Abe and his family, don't get bogged down by the disease. You all are reaching for the stars and I see some shimmer of hope that there is a treatment option coming soon if it is getting approved by the FDA.
Dear Professor, I am trying to implement the work done by Suyu Liu, “A Bayesian Phase I/II Trial Design for Immunotherapy”, using R, since the code attached with that work takes a lot of time (more than 20 hours and the code not complete, I do not know how it produced the tables and the figures). So that I tried to use trialr package trying to get similar or approximate results using the utility functions for sensitivity analysis in table 1 (picture below), but this package allowed me to use just two outcomes ( toxicity, efficacy ) and the work of Liu used three outcomes (immune response, toxicity, and efficacy). I want to see if I used the correct utility (table 1 below) and to see how to add a third outcome ( immune response ) to the model? (Question 1) I attached to you the code and the output for 50 iterations and 60 patients from the work of Liu and Yuan ( I cannot do more, it took around 12 hours), if you know how he produced the results, (Question 2) I hope you can feed me back. My goal is: to use their idea to select the best dose in the first stage and to continue in a second stage with only 2 arms clinical trial and the best dose. (may there is another way to do that?) Table 1 ### My code trying to get similar results using trialr package ### rm(list = ls()) library(trialr) ## Utility from table 1 and Liu and Yuan work. Uti <- array(0,c(2,3,2)) # order: tox, eff, immuno Uti[,,1] <- matrix(c(0,0,50,10,80,35),nrow=2) Uti[,,2] <- matrix(c(5,0,70,20,100,45),nrow=2) N.max= 60 # patients outcomes <- '1NNN 2NNT 3NNT 4NNN 5NTN' doses = c(.1,.3,.5,.7,.9) fit <- stan_efftox(outcomes, real_doses =doses, efficacy_hurdle = 0.5, toxicity_hurdle = 0.3, p_e = 0.1, p_t = 0.1, eff0 = 0.5, tox1 = 0.65, eff_star = 0.7, tox_star = 0.25, alpha_mean = -7.9593, alpha_sd = 3.5487, beta_mean = 1.5482, beta_sd = 3.5018, gamma_mean = 0.7367, gamma_sd = 2.5423, zeta_mean = 3.4181, zeta_sd = 2.4406, eta_mean = 0, eta_sd = 0.2, psi_mean = 0, psi_sd = 1, seed = 123) ndoses <- length(fit$prob_tox) plot(1:ndoses, fit$prob_tox, type="b", pch=19, xlab="Dose level", ylab="Probability of toxicity", ylim=c(0,max(fit$prob_tox) + 0.15), col="green") points(1:ndoses,fit$prob_eff, type="b", pch=18, col="blue") abline(h=0.3, lwd=2, lty=4, col = "red") legend(1, 0.4, legend=c("Toxicity", "Effecacy"), col=c("green", "blue"), lty=1:2, cex=0.8) this produce this figure could you help me even by the paid consultancy? Thanks in advance
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Thanks for a very detailed explanation especially for folks with no background. Q1: for covid what phases were bypassed to expedite the process ? Q2: Are IRB members staff of FDA ? Q3: who funds them ? The Sponsor ?
Q1: for covid what phases were bypassed to expedite the process ? = Depends on case by case. This is not applicable for all engaged in Covid. For example, Moderna was given that choice but others were not (Inovio Pharma) Q2: Are IRB members staff of the FDA ? Yes, its independent body specifically for all Ethical adherence Q3: who funds them ? The Sponsor ? Yes Sponsors
