Enzyme Replacement Therapy for Sanfilippo Syndrome Type B with Dr. Javier Szwarcberg Spruce Biosciences
Release Date: 05/11/2026
Enzyme Replacement Therapy for Sanfilippo Syndrome Type B with Dr. Javier Szwarcberg Spruce Biosciences TRANSCRIPT
Empowered Patient Podcast
Dr. Javier Szwarcberg, CEO of Spruce Biosciences, is developing drugs for rare diseases with a primary focus on Sanfilippo Syndrome Type B, a devastating genetic neurodegenerative disease affecting children. The source of this condition is a lack of an enzyme, which results in a buildup of a toxic substance in the brain. The company's drug is an enzyme replacement therapy administered directly to the brain to bypass the blood-brain barrier, and clinical trial data is showing a strong effect on the toxic substrate in the brain and a meaningful benefit on cognition. Javier...
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Enzyme Replacement Therapy for Sanfilippo Syndrome Type B with Dr. Javier Szwarcberg Spruce Biosciences
Empowered Patient Podcast
Dr. Javier Szwarcberg, CEO of Spruce Biosciences, is developing drugs for rare diseases with a primary focus on Sanfilippo Syndrome Type B, a devastating genetic neurodegenerative disease affecting children. The source of this condition is a lack of an enzyme, which results in a buildup of a toxic substance in the brain. The company's drug is an enzyme replacement therapy administered directly to the brain to bypass the blood-brain barrier, and clinical trial data is showing a strong effect on the toxic substrate in the brain and a meaningful benefit on cognition. Javier...
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AI in Complex Healthcare Revenue Cycle Management with Zach Shultz EnableComp TRANSCRIPT
Empowered Patient Podcast
Zach Shultz, Senior Director of Product Policy and Solutions at EnableComp, describes the challenges of revenue cycle management in healthcare with a focus on the strategic importance of full-time equivalent (FTE) allocation. Hospitals could improve their bottom line by outsourcing complex, low-reimbursement claims, such as those from the VA so that in-house staff can focus on higher-value commercial claims. Despite the emergence of AI as a significant factor in automating the review process, human intervention is irreplaceable for quality assurance, managing escalations, and resolving...
info_outline
AI in Complex Healthcare Revenue Cycle Management with Zach Shultz EnableComp
Empowered Patient Podcast
Zach Shultz, Senior Director of Product Policy and Solutions at EnableComp, describes the challenges of revenue cycle management in healthcare with a focus on the strategic importance of full-time equivalent (FTE) allocation. Hospitals could improve their bottom line by outsourcing complex, low-reimbursement claims, such as those from the VA so that in-house staff can focus on higher-value commercial claims. Despite the emergence of AI as a significant factor in automating the review process, human intervention is irreplaceable for quality assurance, managing escalations, and resolving...
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Empowered Patient Podcast
Lon Hecht, CEO of Care2U, has designed an innovative model of providing in-home high-acuity care as an alternative to emergency room visits or hospitalizations. The process includes sending a clinician to the home within 2-4 hours, with extensive mobile medical equipment, and physician support via telehealth. Benefits include better patient recovery outcomes, significant cost savings for patients and payers, and prevention of costly hospital readmissions. Lon explains, "My company, Care2U, and I are bringing high acuity care into the home. So think of it as kind of a...
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In-Home High-Acuity Care is Transforming Emergency Medicine with Lon Hecht Care2U
Empowered Patient Podcast
Lon Hecht, CEO of Care2U, has designed an innovative model of providing in-home high-acuity care as an alternative to emergency room visits or hospitalizations. The process includes sending a clinician to the home within 2-4 hours, with extensive mobile medical equipment, and physician support via telehealth. Benefits include better patient recovery outcomes, significant cost savings for patients and payers, and prevention of costly hospital readmissions. Lon explains, "My company, Care2U, and I are bringing high acuity care into the home. So think of it as kind of a...
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How Healthcare Organizations Use AI to Transform Patient Access and Revenue Cycle Management with Patty Hayward Talkdesk TRANSCRIPT
Empowered Patient Podcast
Patty Hayward, General Manager of healthcare and life sciences at Talkdesk, describes how a hybrid model of humans and AI is reshaping healthcare contact centers. Patient access and revenue cycle management are the primary, interconnected challenges for these centers, and modern large-language AI is being used to handle complex scheduling logic. Data analytics helps identify patterns in patient inquiries, predict demand peaks, and move care from reactive to proactive. The expectation is that AI agents will guide patients, becoming a normal and accepted part of healthcare. Patty explains, "I...
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How Healthcare Organizations Use AI to Transform Patient Access and Revenue Cycle Management with Patty Hayward Talkdesk
Empowered Patient Podcast
Patty Hayward, General Manager of healthcare and life sciences at Talkdesk, describes how a hybrid model of humans and AI is reshaping healthcare contact centers. Patient access and revenue cycle management are the primary, interconnected challenges for these centers, and modern large-language AI is being used to handle complex scheduling logic. Data analytics helps identify patterns in patient inquiries, predict demand peaks, and move care from reactive to proactive. The expectation is that AI agents will guide patients, becoming a normal and accepted part of healthcare. Patty explains, "I...
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Virtual Reality Therapy for Lazy Eye More Effective Than Traditional Patching with Scott Xiao Luminopia TRANSCRIPT
Empowered Patient Podcast
Scott Xiao, Co-Founder and CEO of Luminopia, is developing a novel, FDA -cleared VR-based treatment for neurovisual disorders with a primary focus on amblyopia, lazy eye, in children aged 4- 12 years old. This approach uses popular TV shows to create an engaging, effective therapy that trains the eyes to work together and is designed as a replacement for traditional eye patching. Clinical data indicate that the vision improvements from this therapy are durable and that the VR approach is well-received by children and their parents. Scott explains, "We are pioneering a new class of...
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Virtual Reality Therapy for Lazy Eye More Effective Than Traditional Patching with Scott Xiao Luminopia
Empowered Patient Podcast
Scott Xiao, Co-Founder and CEO of Luminopia, is developing a novel, FDA -cleared VR-based treatment for neurovisual disorders with a primary focus on amblyopia, lazy eye, in children aged 4- 12 years old. This approach uses popular TV shows to create an engaging, effective therapy that trains the eyes to work together and is designed as a replacement for traditional eye patching. Clinical data indicate that the vision improvements from this therapy are durable and that the VR approach is well-received by children and their parents. Scott explains, "We are pioneering a new class of...
info_outlineDr. Javier Szwarcberg, CEO of Spruce Biosciences, is developing drugs for rare diseases with a primary focus on Sanfilippo Syndrome Type B, a devastating genetic neurodegenerative disease affecting children. The source of this condition is a lack of an enzyme, which results in a buildup of a toxic substance in the brain. The company's drug is an enzyme replacement therapy administered directly to the brain to bypass the blood-brain barrier, and clinical trial data is showing a strong effect on the toxic substrate in the brain and a meaningful benefit on cognition.
Javier explains, "It's a devastating, profoundly affecting disease whereby children are typically born normally and born asymptomatic. And over time, they develop an accumulation of a toxic substrate throughout the body, but primarily the brain. And because of that, it eventually results in a fairly young age, usually between two and a half and I would say three and a half, in symptoms starting. That toxic substance builds in throughout the brain and starts affecting initially learning behavior. Sleep patterns are very much affected, whereby children don't sleep at night and sleep during the day, and they're very hyperactive. And sometimes the diagnosis is confused, and there's no clarity as to what is going on with the child, early in the onset of the disease."
"Yes, it's a genetic disease that's inherited. There's a missing enzyme responsible for the metabolism of this toxic substrate, which I talked about, called heparan sulfate. So when heparan sulfate builds up in the brain, it causes inflammation and neurotoxicity, ultimately resulting in neurodegeneration. Think about it as a disease that is very similar to what you end up seeing with Alzheimer's, whereby the toxic substrate in Alzheimer's is between neurons and outside of the actual cell. In this case, in this disease, the toxic substance, which is different than the one that accumulates in Alzheimer's but builds within the neural cells. Yes, so that's very well-known and very well-characterized."
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