RARECast podcast

Player FM - Internet Radio Done Right

28 subscribers

Lagt til ten år siden

Innhold levert av RARECast. Alt podcastinnhold, inkludert episoder, grafikk og podcastbeskrivelser, lastes opp og leveres direkte av RARECast eller deres podcastplattformpartner. Hvis du tror at noen bruker det opphavsrettsbeskyttede verket ditt uten din tillatelse, kan du følge prosessen skissert her https://no.player.fm/legal.

Species Unite

1
Richard (Kudo) Couto: The Hidden Horror Behind a Billion-Dollar Brand 42:18

for 16 dager siden42:18

Spill senere

Lister

Lik

Likt

42:18

“I used to be the largest dairy consumer on the planet. I used to eat so much dairy and meat. The more that I looked into the dairy industry, the more that I saw that it was the singular, most inhumane industry on the planet, that we've all been lied to, including myself, for years. I always believed that the picture on the milk carton, the cow standing next to her calf in the green field with the red barn in the back was true. It’s certainly the complete opposite.” – Richard (Kudo) Couto Richard (Kudo) Couto is the founder of Animal Recovery Mission (ARM), an organization solely dedicated to investigating extreme animal cruelty cases. ARM has led high-risk undercover operations that have resulted in the shutdown of illegal slaughterhouses, animal fighting rings, and horse meat trafficking networks. Recently, they released a damning investigation into two industrial dairy farms outside of Phoenix, Arizona supplying milk to Coca-Cola’s Fairlife brand. What they uncovered was systemic animal abuse, environmental violations, and a devastating betrayal of consumer trust. While Fairlife markets its products as being sourced "humanely," ARM’s footage tells a very different story—one of suffering, abuse, and corporate complicity. Despite the evidence, this story has been largely ignored by mainstream media—likely due to Coca-Cola’s massive influence and advertising dollars.…

RARECast

Del

Serier hjem•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.

543 episoder

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

RARECast

28 subscribers

updated for en dag siden

Del

Serier hjem•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.

543 episoder

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

Alle episoder

RARECast

1
How an Academic Medical Center Helped Change the Landscape for a Rare Disease 22:50

for 1 day siden22:50

22:50

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

1
Improving Outcomes for People with a Set of Rare Cancers 22:09

for 8 dager siden22:09

22:09

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

1
An Effort to Build a Better DMD Gene Therapy 27:32

for 14 dager siden27:32

27:32

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

1
A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder 32:48

for 22 dager siden32:48

32:48

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

1
Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer 25:12

for 29 dager siden25:12

25:12

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

1
Learning to Take His Vitamins 29:50

for 5 weeks siden29:50

29:50

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

1
The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval 24:16

for 6 weeks siden24:16

24:16

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

1
Engineering Skin Bacteria to Be Live Biotherapeutics 28:27

for 7 weeks siden28:27

28:27

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

1
Seeing the Gene and Cell Therapy Translational Divide as an Opportunity 33:58

for 8 weeks siden33:58

33:58

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

1
My Mother, Myself, and ALS 27:47

for 9 weeks siden27:47

27:47

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

1
From Immovable Object to Advocacy Force 24:11

for 10 weeks siden24:11

24:11

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

1
A Rapid and Scalable Approach for Screening Personalized ASOs 23:56

for 11 weeks siden23:56

23:56

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

1
Addressing the Disease Mechanism of a Rare Kidney Disease 25:52

for 12 weeks siden25:52

25:52

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.…

RARECast

1
The Mother of a Son with SCD, Applies Direct Experience to Her Clinical Trials Work 38:42

for 13 weeks siden38:42

38:42

When Katrina Rice’s son was diagnosed with the rare, hematological disorder sickle cell disease, she got to spend a lot of time understanding how a rare disease affects the daily life of people with a condition and their caregivers. Her son, now 28, has become a participant in clinical trials, and that’s further broadened Rice’s perspective as chief delivery officer of biometrics services at the clinical trial data management and analytics company eClinical Solutions. We spoke to Rice about sickle cell disease, how her experience as a mother of a child with a rare disease has informed her professional life, and how she has used that to educate her colleagues and clinical trial sponsors about how to better engage with rare disease clinical trial participants.…

RARECast

1
A First for Rett Syndrome with More in the Pipeline 23:07

for 14 weeks siden23:07

23:07

In 2023, Acadia Pharmaceuticals won approval for Daybue, the first and only treatment for the rare, neurodevelopmental disorder Rett syndrome. Daybue is not a cure and Acadia continues to pursue additional therapies for the condition including an antisense oligonucleotide therapy to upregulate a protein people with Rett syndrome lack. We spoke to Elizabeth Thompson, executive vice president and head of research and development for Acadia, about Rett syndrome, how Daybue has changed the treatment landscape for people with the condition, and the company’s advancing pipeline to treat it.…

Velkommen til Player FM!

Player FM scanner netter for høykvalitets podcaster som du kan nyte nå. Det er den beste podcastappen og fungerer på Android, iPhone og internett. Registrer deg for å synkronisere abonnement på flere enheter.