RARECast podcast

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Biscuits & Jam

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Encore: Jessica B. Harris Believes in a Welcome Table 42:14

8 hari yang lalu42:14

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Episode Description: Jessica B. Harris may have been born and raised in New York City, but she has Tennessee roots through her father and has spent much of her life split between homes in the Northeast and the South – specifically New Orleans. For more than fifty years, she has been a college professor, a writer, and a lecturer, and her many books have earned her a reputation as an authority on food of the African Diaspora, as well as a lifetime achievement award from the James Beard Foundation. A few years back, Netflix adapted her book, High on the Hog: A Culinary Journey from Africa to America , into a 4 part docuseries. And I’m very proud to say that she’s a longtime contributor to Southern Living with a regular column called The Welcome Table. This episode was recorded in the Southern Living Birmingham studios, and Sid and Jessica talked about her mother’s signature mac and cheese, the cast-iron skillet she’d be sure to save if ever her house were on fire, and her dear friend, the late New Orleans chef Leah Chase. For more info visit: southernliving.com/biscuitsandjam Biscuits & Jam is produced by : Sid Evans - Editor-in-Chief, Southern Living Krissy Tiglias - GM, Southern Living Lottie Leymarie - Executive Producer Michael Onufrak - Audio Engineer/Producer Jeremiah McVay - Producer Learn more about your ad choices. Visit podcastchoices.com/adchoices…

RARECast

Beranda serial•Feed

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

545 episode

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

RARECast

28 subscribers

updated 6 hari yang lalu

Beranda serial•Feed

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

545 episode

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

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RARECast

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Using CRISPR to Modulate Gene Expression 26:10

6 hari yang lalu26:10

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Epicrispr Biotechnologies is using CRISPR to modulate the expression of disease-causing genes without making cuts to DNA. Its lead program is in development to treat FSHD, a genetic disorder that causes progressive weakness in the muscles of the face, shoulders, and upper arms. We spoke to Amber Salzman, CEO of Epicrispr, about how its one-and-done therapies work to target the epigenome, the company’s lead program in FSHD, and the broader applications for its therapeutic approach.…

RARECast

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Satisfying the Hunger for a Prader-Willi Therapy 20:14

13 hari yang lalu20:14

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One of the hallmarks of the rare genetic disorder Prader-Willi syndrome is hyperphagia, an insatiable desire to eat. Households with someone with Prader-Willi syndrome often have to take measures to restrict access to food, such as locking refrigerators, pantries, and trash cans. They must also deal with the complex behavioral challenges associated with the condition. Earlier this year, Soleno Therapeutics won approval for VykatXR, the first FDA-approved therapy for treating hyperphagia. We spoke to Anish Bhatnagar, CEO of Soleno Therapeutics, about Prader-Willi syndrome, its therapy to treat the condition, and the significance of having a medicine that can address hyperphagia associated with the disorder.…

RARECast

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How an Academic Medical Center Helped Change the Landscape for a Rare Disease 22:50

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CTX is a rare metabolic disease that can cause seizures, developmental delays, and intellectual disability. Now, a diagnostic test that can detect the condition early is available, and a treatment that can prevent the disease's serious effects has won regulatory approval. We spoke to Bart Duell, professor of medicine at Oregon Health Sciences University, about CTX, the role OHSU played in developing a diagnostic and advancing a treatment for CTX; and the critical interplay between academic medical centers, patient advocates, and drug developers to address the needs of people with rare diseases.…

RARECast

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Improving Outcomes for People with a Set of Rare Cancers 22:09

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Biliary tract cancers are a group of rare cancers with limited treatment options and generally poor outcomes because most patients are diagnosed at an advanced stage. There are efforts to address that through the development of early detection tools. And the use of biomarker testing can today match as many as half of these patients to a targeted therapy, although it’s critical to make people aware of the importance of this. We spoke to Rachna Shroff, associate director of clinical investigations of the gastrointestinal clinical research team at the University of Arizona Cancer Center, about biliary tract cancers, the importance of biomarker testing, and what can be done to improve outcomes for people with these rare cancers.…

RARECast

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An Effort to Build a Better DMD Gene Therapy 27:32

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Drug development activities focused on the rare, neuromuscular condition Duchenne muscular dystrophy have translated into functional improvements and an extension of life expectancy. Regenxbio is among several companies pursuing a gene therapy to treat Duchenne. Regenxbio believes both its microdystrophin—a truncated form of the dystrophin gene small enough to fit in a vector—and the vector it is using, give it a competitive advantage over other efforts. We spoke Curran Simpson, CEO of Regenxbio, about the company’s platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.…

RARECast

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A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder 32:48

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GRIN-related disorders are a group of rare neurodevelopmental conditions that can cause intellectual disability, behavioral challenges, and seizures. GRIN Therapeutics is developing a once-failed development candidate for chronic neuropathic pain, known as radiprodil, as a potential treatment for GRIN-related disorders driven by gain-of-function mutations. We spoke to Bruce Leuchter, president and CEO of GRIN Therapeutics, about GRIN-related disorders, how the company came to develop radiprodil, and how it represents an effort by Neurvati Neurosciences to bring a portfolio approach to developing novel treatments for neurological and psychiatric disorders.…

RARECast

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Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer 25:12

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Osteosarcoma is a rare and aggressive bone cancer that usually affects children and teenagers. Over the past 40 years, there’s been little progress to improve survival rates, with the five-year survival rate stagnant at about 60 to 70 percent for localized disease and 15 to 30 percent for metastatic osteosarcoma. OS Therapies is developing an experimental immunotherapy to treat certain osteosarcomas. We spoke to Paul Romness, president and CEO of OS Therapies, about why osteosarcomas have been difficult to treat, the company’s experimental therapy for the condition, and the promising results that have been seen to date.…

RARECast

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Learning to Take His Vitamins 29:50

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When Paul Biderman was born, he had trouble gaining weight. He grew sicker and, at about six months, he was diagnosed with the ultra-rare genetic disease abetalipoproteinemia, a condition that impairs a person’s ability to absorb fats and fat-soluble vitamins. The condition requires adherence to a strict diet and a regimen of daily vitamin supplementation, which he didn’t always follow. Now approaching 60, Biderman suffers from neuropathy in his lower limbs and is losing his eyesight. We spoke to Biderman, chief operating officer and patient liaison for the ABL+ Foundation, about living with ABL, the challenges of obtaining adequate nutrition, and the impact becoming a patient advocate has had on him.…

RARECast

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The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval 24:16

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Spinocerebellar ataxia includes a group of rare, genetic, neurodegenerative disorders. People with the condition suffer from the loss of balance, coordination, and muscle control. As the condition progresses, people can lose the ability to walk and speak. There is no approved treatment for SCA, but Biohaven has applied to the U.S. Food and Drug Administration to begin marketing its experimental therapy troriluzole, which can normalize levels of glutamate, a key neurotransmitter implicated in the disease. We spoke to Melissa Beiner, senior medical director at Biohaven, about spinocerebellar ataxia, the company’s therapy under review at the FDA to treat the condition, and why it may have the potential to benefit a number of other neurological conditions as well.…

RARECast

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Engineering Skin Bacteria to Be Live Biotherapeutics 28:27

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Netherton syndrome is a rare and chronic skin condition. One in ten infants with the disease will die as a result of infections and related complications. Azitra has developed a platform for engineering bacteria that naturally colonize the skin to produce proteins with therapeutic benefits to treat Netherton syndrome and other conditions. We spoke to Travis Whitfill, co-founder and chief operating officer of Azitra, about Netherton syndrome, the company’s platform technology for engineering live biotherapeutic products, and why it takes more than simply inserting a gene into a desired bacteria to produce these treatments.…

RARECast

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Seeing the Gene and Cell Therapy Translational Divide as an Opportunity 33:58

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Emerging cell and gene therapies represent areas of great promise for people with rare, genetic diseases, but the translation gap for these medicines can leave them stalled at the lab. Mass General Brigham in 2022 launched the Gene and Cell Therapy Institute, a research hub dedicated to advancing gene and cell therapies for various diseases to bridge the divide between academic labs and clinical development of therapies. The institute brings together more than 500 researchers and clinicians and boasts unique assets, such as its RNA Therapeutics Core, which enable it to produce cutting-edge circular RNA on-site. We spoke to Nathan Yozwiak, head of research at the Mass General Brigham Gene andCell Therapy Institute, about the need it is seeking to address, how the institute operates, and what it might do to enable the development of bespoke therapies for ultra-rare diseases.…

RARECast

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My Mother, Myself, and ALS 27:47

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Raziel Green lost both her mother and aunt to the rare neurodegenerative disease amyotrophic lateral sclerosis or ALS. But when Green, a runner, began experiencing muscle weakness and falls, doctors failed to recognize that she had a genetic form of the condition. Two years after she first sought care, she was diagnosed with the SOD1 form of the disease and enrolled in a clinical trial of what would become the antisense oligonucleotide Qalsody. We spoke to Green about her experience with ALS, her decision to enroll in a clinical trial, and her health more than eight years after her diagnosis.…

RARECast

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From Immovable Object to Advocacy Force 24:11

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While traveling in Australia, Russ Walter was taken to the hospital and treated for heart palpitations. When he returned home, he soon lost the ability to walk. He was diagnosed with the rare autoimmune condition Guillian-Barre syndrome and spent three months in intensive care and rehabilitation. It would take nearly a year of physical therapy before he could walk again. We spoke to Walter, liaison and board member of the GBS-CIDP Foundation International, about his recovery from Guillian-Barre syndrome, the changing medical landscape for the condition, and why his experience led him to become a patient advocate…

RARECast

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A Rapid and Scalable Approach for Screening Personalized ASOs 23:56

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Organoids, three-dimensional cell models that can replicate an individual’s organs, are valuable tools for testing medicines that might treat their illness. It can, however, take up to $10,000 and a year to grow organoids using conventional methods from patient-derived induced pluripotent stem cells. Researchers at Children’s Mercy Kansas City’s Genomic Medicine Center developed a way to do this from about $200 and in two to three weeks. We spoke to Scott Younger, director of disease gene engineering at Children Mercy Kansas City’s Genomic Medicine Center, about the process, the test it ran to match three children with Duchenne muscular dystrophy to an antisense oligonucleotide therapy, and the potential impact this may have on developing customized therapies for people with rare genetic diseases.…

RARECast

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Addressing the Disease Mechanism of a Rare Kidney Disease 25:52

15 weeks yang lalu25:52

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IgA nephropathy is a rare autoimmune kidney disease that can lead to the loss of kidney function. Though a number of treatment options exist today, none address the root cause of the disease and instead seek to manage symptoms, slow progression, or prevent complications. Vera Therapeutics’ experimental fusion protein atacicept, in a phase 2B study, showed the ability to provide long-term kidney function stabilization. We spoke to Marshall Fordyce, founder and CEO of Vera Therapeutics, about IgA nephropathy, atacicept, and its potential in other B cell-mediated diseases.…

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