Novel Precision Gene Therapy for Friedreich's Ataxia with Dr. Gabriel Brooks Solid Biosciences

Dr. Patricia Hayes is the Chief Medical Officer of Imagine Pediatrics, which has developed a hybrid care model for children with special needs.  Imagine Pediatrics provides 24/7 virtual and in-home care, which integrates medical, behavioral, social, and pharmacy services to support families and existing providers and help prevent unnecessary emergency room visits. The organization uses technology and predictive models to identify at-risk children and proactively target interventions to increase the number of safe days at home for children with complex conditions. Patricia explains, "Imagine Pediatrics is a 24/7, 365 virtual and in- home care for children and youth with special health care needs and medical complexity. We offer integrated medical, behavioral, and social services, inclusive of also pharmacy services, social services, as I mentioned, behavioral health therapy. It's just really an amazing form of wraparound care supporting these kids and supporting the providers that care for them outside of Imagine Pediatrics as well."   "We are in multiple states, and we can provide those services in the states where we're practicing from border to border. So because we're virtual first, we're able to see kids in rural areas, urban areas, wherever they may be, and offer in-home services to them as well when they may have any sort of difficulty, either need additional services that we can't provide virtually, or they may have difficulty going to an emergency room or difficulty seeing their primary care pediatrician or their specialist. We could offer that support in home as well." "It's a very wide spectrum. We have anywhere from children who have extreme medical complexity, who may be dependent on equipment, who may have a tracheostomy, be ventilated, have a G-tube, all the way to children who may just have severe asthma or be seen and utilize the hospital quite a bit for their asthma. See a wide range of behavioral health conditions as well, anywhere from severe depression to bipolar or anxiety, and early schizophrenia. And so it's an extremely wide range of really our care that we provide." #ImaginePediatrics #pediatrics #PediatricHealthcare #ComplexCare #ValueBasedCare #VBC #PediatricCare #HealthcareInnovation #VirtualHealth #SpecialNeeds #ChildHealth #HealthTech #PatientCare #MedicalHome #HealthcareAccess #PediatricMedicine #TeleHealth #IntegratedCare Imaginepediatrics.org Listen to the podcast here

Dr. Patricia Hayes is the Chief Medical Officer of Imagine Pediatrics, which has developed a hybrid care model for children with special needs.  Imagine Pediatrics provides 24/7 virtual and in-home care, which integrates medical, behavioral, social, and pharmacy services to support families and existing providers and help prevent unnecessary emergency room visits. The organization uses technology and predictive models to identify at-risk children and proactively target interventions to increase the number of safe days at home for children with complex conditions. Patricia explains, "Imagine Pediatrics is a 24/7, 365 virtual and in- home care for children and youth with special health care needs and medical complexity. We offer integrated medical, behavioral, and social services, inclusive of also pharmacy services, social services, as I mentioned, behavioral health therapy. It's just really an amazing form of wraparound care supporting these kids and supporting the providers that care for them outside of Imagine Pediatrics as well."   "We are in multiple states, and we can provide those services in the states where we're practicing from border to border. So because we're virtual first, we're able to see kids in rural areas, urban areas, wherever they may be, and offer in-home services to them as well when they may have any sort of difficulty, either need additional services that we can't provide virtually, or they may have difficulty going to an emergency room or difficulty seeing their primary care pediatrician or their specialist. We could offer that support in home as well." "It's a very wide spectrum. We have anywhere from children who have extreme medical complexity, who may be dependent on equipment, who may have a tracheostomy, be ventilated, have a G-tube, all the way to children who may just have severe asthma or be seen and utilize the hospital quite a bit for their asthma. See a wide range of behavioral health conditions as well, anywhere from severe depression to bipolar or anxiety, and early schizophrenia. And so it's an extremely wide range of really our care that we provide." #ImaginePediatrics #pediatrics #PediatricHealthcare #ComplexCare #ValueBasedCare #VBC #PediatricCare #HealthcareInnovation #VirtualHealth #SpecialNeeds #ChildHealth #HealthTech #PatientCare #MedicalHome #HealthcareAccess #PediatricMedicine #TeleHealth #IntegratedCare Imaginepediatrics.org Download the transcript here

Shashi Shankar, CEO of Novellia,  focuses on enabling patients with serious medical conditions to consolidate their medical history into a single record using a free app.  The business model provides anonymized real-world data to pharmaceutical companies for research, clinical trial recruitment, and the acceleration of new therapy development. Artificial intelligence is used to help patients make sense of their fragmented medical records, identify clinical trials, and better understand drug safety profiles and therapeutic effectiveness.  Shashi explains, "In a nutshell, what we do is we help folks living with serious and complex medical conditions find access to and then collect all of their medical records going back 20 plus years across different physicians, sites of care, labs, and insurance into one always continuously updated record that then helps them manage and navigate their course of care." "I actually spent months of my career working at Genentech and Roche, working on a number of different cancer therapeutics, and it was an amazing opportunity, and you get to meet so many different patient communities. But one of the most difficult parts, I think, for anybody who's working on developing new medications and new therapies, is that you need to really understand the communities that you hope to serve one day. And unfortunately, a lot of data that exists right now doesn't actually capture the true patient journey. And it's not very representative of the communities that are dealing with and trying to navigate all of these different conditions." "The benefit of being able to share this data with researchers in a way that's anonymous and de- identified, so patient data is always totally secure and always held private by Novellia. The benefit of this is that researchers are able to finally understand the full journey of patients and think through how their therapies can actually help those who are trying to navigate their care."  #Novellia#HealthcareInnovation #PatientEmpowerment #DigitalHealth #HealthTech #MedicalRecords #AIinHealthcare #PatientCenteredCare #HealthcareData #ClinicalTrials #Oncology #PharmaceuticalResearch #HealthIT Novellia.com Listen to the podcast here

Shashi Shankar, CEO of Novellia,  focuses on enabling patients with serious medical conditions to consolidate their medical history into a single record using a free app.  The business model provides anonymized real-world data to pharmaceutical companies for research, clinical trial recruitment, and the acceleration of new therapy development. Artificial intelligence is used to help patients make sense of their fragmented medical records, identify clinical trials, and better understand drug safety profiles and therapeutic effectiveness.  Shashi explains, "In a nutshell, what we do is we help folks living with serious and complex medical conditions find access to and then collect all of their medical records going back 20 plus years across different physicians, sites of care, labs, and insurance into one always continuously updated record that then helps them manage and navigate their course of care." "I actually spent months of my career working at Genentech and Roche, working on a number of different cancer therapeutics, and it was an amazing opportunity, and you get to meet so many different patient communities. But one of the most difficult parts, I think, for anybody who's working on developing new medications and new therapies, is that you need to really understand the communities that you hope to serve one day. And unfortunately, a lot of data that exists right now doesn't actually capture the true patient journey. And it's not very representative of the communities that are dealing with and trying to navigate all of these different conditions." "The benefit of being able to share this data with researchers in a way that's anonymous and de- identified, so patient data is always totally secure and always held private by Novellia. The benefit of this is that researchers are able to finally understand the full journey of patients and think through how their therapies can actually help those who are trying to navigate their care."  #Novellia#HealthcareInnovation #PatientEmpowerment #DigitalHealth #HealthTech #MedicalRecords #AIinHealthcare #PatientCenteredCare #HealthcareData #ClinicalTrials #Oncology #PharmaceuticalResearch #HealthIT Novellia.com Download the transcript here

Dr. Gabriel Brooks is Chief Medical Officer at Solid Biosciences, a precision molecular genetic medicines company focused on rare cardiovascular and neuromuscular diseases, including Friedreich's ataxia. Currently, there are very limited treatments for this rare, progressive neurologic disease caused by a genetic deficiency. Solid Biosciences' novel gene therapy uses dual-route administration to deliver directly to the heart and brain and to replace the missing frataxin gene, which is critical for energy production. Dr. Brooks explains, "Our flagship program is our DMD program, where we have two clinical trials, a first-in-human INSPIRE study, and a double-blind randomized placebo-controlled phase three trial for the SGT-003 DMD medicine. And for Friedreich's ataxia, we have the SGT-212 program, which uses a novel dual route of administration to target not only the cardiomyopathy, but also uses direct injection into the dentate nucleus. We're trying to address the central pathophysiology of the ataxia that patients live with every day." "So Friedrich's ataxia is a rare and devastating neurologic disease that afflicts around 5,000 patients in the United States and much more actually in Europe. There is a genetic predisposition. And Friedrich's ataxia is really a disease where the patients experience difficulty in moving, what's called ataxia, which you could think of as poor coordination, where when they try to move, their brain is sending a signal to their muscles, let's say to grab that cup of coffee or climb the stairs." "In fact, there are specific neurologic tests in terms of looking at nerve conduction and other things that can make the formal diagnosis. And certainly, we can get there with genetic testing. And what you're picking up on is absolutely something that's important with rare disease, is that oftentimes there's a lag between when a patient first manifests symptoms and when they ultimately have the diagnosis. In Friedrich's ataxia, like other genetic diseases, it is horrible. And so, for patients who do make the diagnosis of Friedreich's ataxia, there is often what we call cascade screening, where we look for the disease gene in relatives. Oftentimes, it's then that siblings are identified, and eventually they start manifesting the disease as well."   #SolidBiosciences #GeneTherapy #GeneTherapyResearch #RareDiseases #FriedreichsAtaxia #PrecisionMedicine #Neurology #Cardiology #ClinicalTrials #MedicalInnovation #HealthcareInnovation #SolidBiosciences solidbio.com Listen to the podcast here

Dr. Gabriel Brooks is Chief Medical Officer at Solid Biosciences, a precision molecular genetic medicines company focused on rare cardiovascular and neuromuscular diseases, including Friedreich's ataxia. Currently, there are very limited treatments for this rare, progressive neurologic disease caused by a genetic deficiency. Solid Biosciences' novel gene therapy uses dual-route administration to deliver directly to the heart and brain and to replace the missing frataxin gene, which is critical for energy production. Dr. Brooks explains, "Our flagship program is our DMD program, where we have two clinical trials, a first-in-human INSPIRE study, and a double-blind randomized placebo-controlled phase three trial for the SGT-003 DMD medicine. And for Friedreich's ataxia, we have the SGT-212 program, which uses a novel dual route of administration to target not only the cardiomyopathy, but also uses direct injection into the dentate nucleus. We're trying to address the central pathophysiology of the ataxia that patients live with every day." "So Friedrich's ataxia is a rare and devastating neurologic disease that afflicts around 5,000 patients in the United States and much more actually in Europe. There is a genetic predisposition. And Friedrich's ataxia is really a disease where the patients experience difficulty in moving, what's called ataxia, which you could think of as poor coordination, where when they try to move, their brain is sending a signal to their muscles, let's say to grab that cup of coffee or climb the stairs." "In fact, there are specific neurologic tests in terms of looking at nerve conduction and other things that can make the formal diagnosis. And certainly, we can get there with genetic testing. And what you're picking up on is absolutely something that's important with rare disease, is that oftentimes there's a lag between when a patient first manifests symptoms and when they ultimately have the diagnosis. In Friedrich's ataxia, like other genetic diseases, it is horrible. And so, for patients who do make the diagnosis of Friedreich's ataxia, there is often what we call cascade screening, where we look for the disease gene in relatives. Oftentimes, it's then that siblings are identified, and eventually they start manifesting the disease as well."   #SolidBiosciences #GeneTherapy #GeneTherapyResearch #RareDiseases #FriedreichsAtaxia #PrecisionMedicine #Neurology #Cardiology #ClinicalTrials #MedicalInnovation #HealthcareInnovation #SolidBiosciences solidbio.com Download the transcript here

Dr. Payam Zamani, Founder and CEO of MY DR NOW, is directly solving the challenge of access to healthcare by creating a hybrid model that includes convenient walk-in clinics with extended hours, house calls, and virtual visits.  This approach is consistent with modern on-demand expectations and attracting patients in dense urban areas as well as rural communities. Patient acquisition is driven by word-of-mouth and relationships with payers and hospital systems, to improve outcomes and reduce hospital readmissions. The model is also attractive to physicians by minimizing administrative tasks and flexible scheduling, leading to less burnout and higher retention. Payam explains, "The big issue with healthcare, as we've seen it and that MY DR NOW has taken on, one of the most challenging issues of healthcare is accessibility. For far too long, there's been this discrepancy between what we believe and what we know as healthcare practitioners to be important versus reality, versus what actually happens. We have a tendency to focus on all the right things and give patients all the right instructions and directions, as far as focusing on prevention, cancer screenings, and annual wellness visits. And we tell them to manage their chronic conditions because it's important, and to take their medications. But in reality, what ends up happening is that patients have to take time off work just to be able to see their healthcare provider. They have to wait weeks or, in many instances, months in advance just to be able to get in to see someone."   "What MY DR NOW really set out to solve is how to deliver healthcare services in a manner that's more consistent with our daily lives, with what our expectations are. In a manner that makes more sense for working people, for working families, for moms and kids and for parents, and in a way that delivers better outcomes. And that's MY DR NOW in a nutshell." "Well, along those same lines of accessibility and convenience, what we set out to do was first of all, build an omnichannel network. And what that means is having multiple modalities by which you can obtain healthcare services. So there are clinics, and we like to make sure that those clinics are ubiquitous, and we can expand on that here in a little bit. But we have clinics that are open seven days a week. They're open early and late, on weekends and holidays. They're open when you expect them and need them to be open. But more importantly, you can not only schedule an appointment for those clinics, but also walk in whenever you want. And just as importantly, you can walk in for anything you go to your family medicine office for. So it's not an urgent care where you're only going in for acute and episodic issues."   #MYDRNOW #HealthcareInnovation #PrimaryCare #PatientCentered #HealthcareAccessibility #DigitalHealth #ValueBasedCare #HealthTech #PhysicianWellbeing #HealthcareDelivery #MedicalInnovation #AccessToCare #HealthcareInnovation #OmnichannelCare #HealthcareTransformation mydrnow.com Listen to the podcast here  

Dr. Payam Zamani, Founder and CEO of MY DR NOW, is directly solving the challenge of access to healthcare by creating a hybrid model that includes convenient walk-in clinics with extended hours, house calls, and virtual visits.  This approach is consistent with modern on-demand expectations and attracting patients in dense urban areas as well as rural communities. Patient acquisition is driven by word-of-mouth and relationships with payers and hospital systems, to improve outcomes and reduce hospital readmissions. The model is also attractive to physicians by minimizing administrative tasks and flexible scheduling, leading to less burnout and higher retention. Payam explains, "The big issue with healthcare, as we've seen it and that MY DR NOW has taken on, one of the most challenging issues of healthcare is accessibility. For far too long, there's been this discrepancy between what we believe and what we know as healthcare practitioners to be important versus reality, versus what actually happens. We have a tendency to focus on all the right things and give patients all the right instructions and directions, as far as focusing on prevention, cancer screenings, and annual wellness visits. And we tell them to manage their chronic conditions because it's important, and to take their medications. But in reality, what ends up happening is that patients have to take time off work just to be able to see their healthcare provider. They have to wait weeks or, in many instances, months in advance just to be able to get in to see someone."   "What MY DR NOW really set out to solve is how to deliver healthcare services in a manner that's more consistent with our daily lives, with what our expectations are. In a manner that makes more sense for working people, for working families, for moms and kids and for parents, and in a way that delivers better outcomes. And that's MY DR NOW in a nutshell." "Well, along those same lines of accessibility and convenience, what we set out to do was first of all, build an omnichannel network. And what that means is having multiple modalities by which you can obtain healthcare services. So there are clinics, and we like to make sure that those clinics are ubiquitous, and we can expand on that here in a little bit. But we have clinics that are open seven days a week. They're open early and late, on weekends and holidays. They're open when you expect them and need them to be open. But more importantly, you can not only schedule an appointment for those clinics, but also walk in whenever you want. And just as importantly, you can walk in for anything you go to your family medicine office for. So it's not an urgent care where you're only going in for acute and episodic issues."   #MYDRNOW #HealthcareInnovation #PrimaryCare #PatientCentered #HealthcareAccessibility #DigitalHealth #ValueBasedCare #HealthTech #PhysicianWellbeing #HealthcareDelivery #MedicalInnovation #AccessToCare #HealthcareInnovation #OmnichannelCare #HealthcareTransformation mydrnow.com Download the transcript here

Tom Kim, Chief Medical Officer at Sound Long-Term Care Management, discusses the role of accountable care organizations, ACOs, in improving healthcare for the long-term care population. The unique complexities and challenges of these patients include multiple medical conditions, frequent hospitalizations, and fragmented care.  By using predictive analytics, telemedicine, and value-based care principles to deliver more coordinated care, patients are avoiding hospitalizations, receiving higher-quality care, and experiencing reduced costs. Tom explains, "So, in essence, an accountable care organization (ACO) is a group of healthcare providers that come together with a commitment to Medicare to improve the quality of care for the Medicare beneficiaries they serve and to decrease the cost of care for that population. And it's a way to really better coordinate care for those residents or for those Medicare beneficiaries to really receive the best outcome possible."   "Yes, so in the fee-for-service world, there is a lot of focus, at least from a provider standpoint, on volume. So you're really focused on seeing as many patients as you can. In a value-based care model, we're really moving away from the volume model toward one focused more on quality. So, instead of being rewarded for high volume, you're being rewarded as a provider for the quality of care you provide. And not only is this beneficial to the provider, but it's also beneficial to the patients. So, there's having more time with their provider, receiving better care, and hopefully avoiding unnecessary or avoidable care. And so it's really a win across the board, from both a provider and a patient standpoint."  "Our patient population tends to be more complex with more medical problems. They tend to be hospitalized more often. They tend to rely on others to make healthcare decisions and closer to the end of life. And so, care is really different from those who are still living at home and in the community. And on top of that, in terms of the long-term care space, and why it's so challenging is that you just kind of mentioned it a little bit, is that the population is aging."   #SLTCM #ACO #AccountableCareOrganizations #AccountableCare #LongTermCare #ValueBasedCare #HealthcareInnovation #Telemedicine #SeniorCare #HealthcareLeadership #PatientCare #HealthTech #Medicare sltcm.soundphysicians.com Listen to the podcast here

Tom Kim, Chief Medical Officer at Sound Long-Term Care Management, discusses the role of accountable care organizations, ACOs, in improving healthcare for the long-term care population. The unique complexities and challenges of these patients include multiple medical conditions, frequent hospitalizations, and fragmented care.  By using predictive analytics, telemedicine, and value-based care principles to deliver more coordinated care, patients are avoiding hospitalizations, receiving higher-quality care, and experiencing reduced costs. Tom explains, "So, in essence, an accountable care organization (ACO) is a group of healthcare providers that come together with a commitment to Medicare to improve the quality of care for the Medicare beneficiaries they serve and to decrease the cost of care for that population. And it's a way to really better coordinate care for those residents or for those Medicare beneficiaries to really receive the best outcome possible."   "Yes, so in the fee-for-service world, there is a lot of focus, at least from a provider standpoint, on volume. So you're really focused on seeing as many patients as you can. In a value-based care model, we're really moving away from the volume model toward one focused more on quality. So, instead of being rewarded for high volume, you're being rewarded as a provider for the quality of care you provide. And not only is this beneficial to the provider, but it's also beneficial to the patients. So, there's having more time with their provider, receiving better care, and hopefully avoiding unnecessary or avoidable care. And so it's really a win across the board, from both a provider and a patient standpoint."  "Our patient population tends to be more complex with more medical problems. They tend to be hospitalized more often. They tend to rely on others to make healthcare decisions and closer to the end of life. And so, care is really different from those who are still living at home and in the community. And on top of that, in terms of the long-term care space, and why it's so challenging is that you just kind of mentioned it a little bit, is that the population is aging."   #SLTCM #ACO #AccountableCareOrganizations #AccountableCare #LongTermCare #ValueBasedCare #HealthcareInnovation #Telemedicine #SeniorCare #HealthcareLeadership #PatientCare #HealthTech #Medicare sltcm.soundphysicians.com Download the transcript here