Magda Granata
Scientific projet manager at Fondation Maladies Rares. I coordinate the development of this and other MOOCs within the European Joint Programme on Rare Diseases.
Location France
Activity
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Thank you @SamyKijner , I can suggest you to contact the support of FutureLearn to communicate the technical problems you are facing: support@futurelearn.com .
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Magda Granata replied to Samy Kijner
Hello @SamyKijner, thank you for your message. We will inform FutureLearn about this, we are not directly managing the platform, we are only responsible of the content of the MOOC.
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Magda Granata replied to Ali Ahmed
Hello @AliAhmed and welcome! Do not hesitate to share your comments/views in the next steps!
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Magda Granata replied to Dr Dhan Raj Bagri
Hello @DrDhanRajBagri and welcome! We hope that you will find this course helpful for your professional interests! Looking forward to hearing from you in the next steps!
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Yes, in case of minor patients, the parents can give informed consent for most medical decisions on behalf of them.
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Hello @SamyKijner , try to reload the page and let us know if it works.
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Thank you once again for your feedback @SamyKijner . Indeed, in the previous runs of this MOOC we had some GPs among the learners who found this course particularly relevant for their profession, as you are saying. We hope to have more and more.
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Thank you for your feedback @SamyKijner ; we developed a glossary in order to have a reference step where anyone could come back in case one term is not know/not clear.
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Hello @SamyKijner , we are sorry you have this problem, we would suggest you to come back to this step later or to reload the page. Unfortunately it does not depend from us.
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Hello @SamyKijner thank you for your feedback, we will try to modify the format of this step; in the meanwhile you may find useful the transcript of the audio immediately before the comments' section.
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Hello @SamyKijner , thank you for sharing your views. For this reason we believe that spreading awareness around RDs at different levels is fundamental.
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Magda Granata replied to Samy Kijner
Hello @SamyKijner , we are sorry you have this problem, we would suggest to reload the page.
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Hello @SamyKijner, we do not have this problem, we would suggest you to check the connection. Let us know.
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Hello @LarsMikkelIsager to what step you are referring to exactly?
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Thank you @FlorenceRICCARDI for pointing out these useful resources!
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Thank you @FlorenceRICCARDI for your positive feedback!
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Thank you @FlorenceRICCARDI for your nice feedback! We will communicate it to Elizabeth!
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Thank you @SaedNuhAhmed for this positive feedback; by the end of the year we will launch another MOOC completely focused on the innovative therapies for rare diseases. Stay tuned! :-)
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Magda Granata replied to Yannick Collé
Hello @EmmaGurashiNikolaaoyM.D and welcome! Looking forward to hearing from you in the next steps!
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Hello @SaedNuhAhmed, we went back to Mike once again and here you are his answer:
Poor drug-like properties contributed to the 30%–40% drug development failures in the 1990s; but they only account for 10%–15% of drug development failures today. The section on 2.3. Select best lead drug candidate with optimal drug-like properties in the article... -
Well noted @SaedNuhAhmed ! Thank you!
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Magda Granata replied to Doris Pinto
Dear @DorisPinto, if you are interested in the RD field, we recommend you also to attend to our MOOC "Diagnosing Rare Diseases: from the clinic to research and back": https://www.futurelearn.com/courses/rare-genetic-disease
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Hello @DorisPinto , thank you for your comment. Indeed, international collaborations and initiatives are key to tackle undiagnosed rare diseases.
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Magda Granata replied to Paulo Gaspar
Hello @PauloGaspar ! looking forward to your comments and feedbacks in the next steps!
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- Usually the formulation properties of a repositioned medicine (if that is what the question refers to) meet the needs of the new indication. In that case the original regulatory package of the manufacturing, PK and preclinical studies can be used, and no further formulation, or preclinical safety work is required. Only clinical testing for the new indication...
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Dear @SaedNuhAhmed we have posed your comments to Mike and here you are his answers:
- Understanding the molecular basis of a disease process is critically important for identifying a potential target. It is rare that the exact mechanism is understood (with the exception of single gene defects). Unfortunately, the literature also contains erroneous data... -
I agree with you @SaedNuhAhmed ! Thank you for your positive comment.
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Magda Granata replied to Saed Nuh Ahmed
Thank you @SaedNuhAhmed for your nice feedback. Please, note that this is an old "run" of the course and a new one was launched two years ago, so if you want to continue the discussion with the other learners I would recommend you to go there (follow the link: https://www.futurelearn.com/courses/introduction-to-translational-research-for-rare-diseases)
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Thank you for your comment @LarsMikkelIsager . This is true for all the diseases, but it’s particularly relevant for rare diseases, where several challenges are met in the clinical trials design: the difficulty in finding patients (due to the low numbers and the geographic dispersion) , the inter-individual variability of the disease, the difficulty in the...
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Thank you for your comment @LarsMikkelIsager , these aspects will be discussed more in details in the next weeks of the MOOC. Do not hesitate to comment and share your opinion with the other learners in the next steps!
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Hello @MinhNguyen-Currie , thank you for your comment; the idea here was to use the case of Drisapersen for Duchenne Dystrophy as an example to understand which aspects should be kept in mind when designing clinical trials, in particular here we highlight the importance of natural history studies and outcome measures.
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Magda Granata replied to Pauline BOURNON
Bonjour Pauline! Welcome here! We hope that this MOOC will be helpful for your professional interests. Do not hesitate to comment in the next steps!
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Thank you for your positive feedback @LarsMikkelIsager !
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Hello @LarsMikkelIsager, we will discuss more in details clinical trials and post-marketing authorization studies respectively in week 4 and 5. Looking forward to your comments there!
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Magda Granata replied to Florence RICCARDI
Welcome Florence! Looking forward to your comments and feedbacks in the next steps!
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Thank you @MinhNguyen-Currie for your nice feddback! Happy that you've appreciated it.
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Hello @SaedNuhAhmed , thank you for your feedback! It's very useful! Indedd we don't speak about umbrella trial design, but we mention surrogate end points in week 4.
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Hello @SaedNuhAhmed, to complete @RomainNicolle answers:
- The retrospective study (NCT03701568) was an observational study that collected and evaluated the records of the patients that were treated under compassionate basis. It collects clinical data from the local clinical centers of the patients before and after they enrolled in the compassionate... -
Hello @SaedNuhAhmed , we transferred your positive feedback to Dr Marti who replied "I really appreciate your comments, and encourage you to further approach this field. Thank you!" :-)
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Hello @MinhNguyen-Currie do not hesitate to point out others, if you know them.
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Thank you for your positive feedback @MinhNguyen-Currie
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Thank you for your positive feedback @SaedNuhAhmed ! We hope you will find interesting also next weeks!
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Thank you @SaedNuhAhmed for your nice feedback. Do you think other terms should be included?
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Magda Granata replied to Minh Nguyen-Currie
Hello @JoelDzenza! I hope you will find this course useful for your professional interests. Do not hesitate to comment/ask questions in the next steps!
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Magda Granata replied to Minh Nguyen-Currie
Hello @MinhNguyen-Currie, welcome. This MOOC is focused on the process of developing treatments for rare diseases. I don't know if the topics discussed perfectly fit with what you are looking for, nonetheless I hope you will find it interesting for you.
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Magda Granata replied to Maritta Löytömäki
Hello @MarittaLöytömäki ! Glad that you are here! Looking forward to your comments and feedback in the next steps!
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Magda Granata replied to Natalia Arruti
Hello @Natalia and welcome here! In this MOOC we will discuss more the translational process that leads to drug development, but we will soon launch another MOOC dealing more in details with the innovative therapies, including gene therapies; so: enjoy this MOOC and stay tuned!
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Magda Granata replied to Ece Erdag
Hello @EceErdag and welcome! I hope you will find this MOOC useful for your professional interests. We will discuss preclinical models in particular in week 3. Enjoy the MOOC!
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Magda Granata replied to Dr Arun S
Hello @DrArunS ! Welcome here! Do not hesitate to share your thoughts and comments in the next steps with the other participants!
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Thank you @AnaVerde for your positive feedback. I invite you (if you are interested) in having a look also at our MOOC on diagnosing rare diseases and in staying tuned since new MOOCs will be launched next year on different aspects of rare diseases' research.
