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PODCAST

Patient Empowerment Program: A Rare Disease Podcast

n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)

Join the nano-rare disease community! Interviews features leading physicians, scientists, biotech experts, and patient advocates. Lessons teach core concepts about drugs. Our host Dr. Crooke has led the creation of antisense technology and his foundation, n-Lorem, is using this powerful technology to discover, develop, and provide personalized experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. n-Lorem is a non-profit organization established to apply the

science life sciences

All Episodes

31:53

Coming Together for the Nano-rare Patient

Patient Empowerment Program: A Rare Disease Podcast ·

2023/05/03

This episode is proudly sponsored by our partner, Alnylam Pharmaceuticals, the leading RNAi therapeutics company. When Stan Crooke ran Ionis and John Maraganore ran Alnylam, they were partners that turned into rivals — and not always friendly ones — as they persevered to pursue an entirely new therapeutic space; RNA-targeted drug discovery and development. Now, the pair of drug discovery titans have united once again in support of Dr. Crooke’s n-Lorem Foundation—working to provide personalized

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51:24

Q&A #1 with Patient Families

Patient Empowerment Program: A Rare Disease Podcast ·

2023/04/19

Amber Freed and Shanna Tolbert join n-Lorem founder and CEO, Stan Crooke, for the first ever Patient Empowerment Program Q&A episode. Amber and Shanna are two nano-rare patient mothers and rare disease advocates who have experience with the n-Lorem pathway to treatment as caregivers. Through their experiences, and by canvasing others in the nano-rare community, they’ve compiled questions aimed to help current and potential n-Lorem patients and their families become more knowledgeable about the

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21:41

Bits and Bytes Help Streamline Bench to Bedside

Patient Empowerment Program: A Rare Disease Podcast ·

2023/04/05

The creation of a drug consists of a copious and complicated series of projects that are all intertwined. Andy Mehrotra, CEO of Unipr, joins the Patient Empowerment Program to discuss how Unipr’s AI based cloud program, project, and portfolio management platforms allow organizations like n-Lorem to quickly analyze data to inform program management. Today, n-Lorem effectively manages more than 90 accepted patient programs thanks to help from Unipr. Discovering locations where productivity can be

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31:53

Fostering Collaboration to Fight ALS

Patient Empowerment Program: A Rare Disease Podcast ·

2023/03/22

Manish Raisinghani joins the Patient Empowerment Program to discuss how Target ALS fosters collaboration between academia and industry to research and accelerate the fight against ALS. Target ALS has funded over 50 collaborative projects. Greater than 50% of which have had an industry partner and over 60% have resulted in an ongoing drug discovery program. From their Innovative Ecosystem, 6 clinical trials have emerged. Target ALS’ commitment to fight all forms of ALS, no matter how rare, is evident

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25:02

The Energy of Life

Patient Empowerment Program: A Rare Disease Podcast ·

2023/03/08

Regardless of the circumstances, life finds a way! Jurassic Park got that one right. Cells need to live and depending on the conditions they will use one of two systems that take nutrients, break them down and use that process to generate energy. That energy is needed to sustain life – hence why it is called The Energy of Life. All the order that you see in the night sky, like planets, star clusters and galaxies, are due to an incredible amount of energy in the universe, creating order out of chaos

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47:08

Everybody Lives

Patient Empowerment Program: A Rare Disease Podcast ·

2023/02/15

Dan Doctoroff joins the Patient Empowerment Program to talk about his mission to support a world where everyone with ALS lives. Dan is the former NYC deputy mayor for economics and former CEO of both Bloomberg L.P. and Sidewalk Labs. In this episode, Dan discusses his family’s battle with amyotrophic lateral sclerosis (ALS), his own inspiring story of how his diagnosis completely changed his outlook on life and his work with the foundation he started, Target ALS.On This Episode We Discuss:Dan’s

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22:23

Cardiovascular System Part 4B: (The Spigot) Kidney...

Patient Empowerment Program: A Rare Disease Podcast ·

2023/02/01

If your car’s oil filter is clogged, it’ll lead to severe issues within your engine. The same goes for the blood filters of your body... but worse.The kidneys filter water-salable waste from the blood. As kidneys fail, waste builds up.A blockage in your kidneys may cause a heart attack, stroke, or kidney failure. Detecting kidney disease early on can save lives! While kidney disease isn't reversible, it can be treated to slow or stop the progression of the disease.n-Lorem is accepting nano-rare

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20:23

Cardiovascular System Part 4A: (The Spigot) Kidney...

Patient Empowerment Program: A Rare Disease Podcast ·

2023/01/18

There is so much filtration around you, you may not even notice. Your water, coffee, air, gasoline, and social media feeds... all filtered. So is your blood! It's just a part of everyday life at this point. This episode of the Patient Empowerment Program focuses on the functions of the spigot of the cardiovascular system – the Kidneys. Their main job? To remove water salable waste from the body along with other functions that are necessary for life. Did we mention that n-Lorem is accepting nano-rare

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24:00

Cardiovascular System Part 3: (The Pipes) Arteries...

Patient Empowerment Program: A Rare Disease Podcast ·

2023/01/04

Drip... Drip. When your pipes are leaking, it's never a good thing. Who you gon’ call, YOUR DOCTOR! The next lesson in our ‘pipe’-line focuses on the pipes of the cardiovascular system - the Arteries and Veins. Learn about the arterial and venous systems that manage blood flows with differing pressures, high and low. However, both systems have mechanisms thatcontrol blood flow under unique conditions to supply our tissues with nutrients, remove waste, and keep us alive. In the event of a busted

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27:59

CROs and Their Role for Nano-rare

Patient Empowerment Program: A Rare Disease Podcast ·

2022/12/14

This episode issponsored by our partner, Parexel, one of the world’s largest clinical research organizations (CROs) providing the full range of Phase I to IV clinical development services. Parexel provides clinical operations, real-world data solutions, medical and regulatory expertise and innovative clinical research tools that significantly enhance and streamline n-Lorem’s therapeutic development efforts. Their depth of industry knowledge and strong track record gained over the past 40 years is

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22:25

Cardiovascular System Part 2B: (The Pump) Heart Diseases

Patient Empowerment Program: A Rare Disease Podcast ·

2022/11/30

In this episode, Stan continues his lecture series on organs, concluding his lesson on the pump of the cardiovascular system, the Heart. Organs are individual tissue components that help meet the needs of an entire organism (that's you). The heart is one of the most important, complex, and fascinating organs in your body - and we couldn't squeeze it into just one episode! Learn all about heart diseases in the final installment of this two-part series.

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20:56

Cardiovascular System Part 2A: (The Pump) Heart Anatomy

Patient Empowerment Program: A Rare Disease Podcast ·

2022/11/16

In this episode, Stan continues his lecture series on organs, focusing on the pump of the cardiovascular system – the Heart. Organs are individual tissue components that help meet the needs of an entire organism (that’s you).The heart is one of the most important, complex, and fascinating organs in your body.Learn all about the heart that’s working so hard for you in the first of a two-part series on this amazing organ.

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35:56

Shifting Mindsets to Expedite Rare Treatments

Patient Empowerment Program: A Rare Disease Podcast ·

2022/11/02

This episode is sponsored by our partner, Ultragenyx, a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases! Ultragenyx has been a loyal and proud supporter of n-Lorem and our patients.Stan talks with a rare disease treatment advocate and the CEO, President and Founder of Ultragenyx, Emil Kakkis, MD, PhD, about why he has dedicated his career to supporting the rare disease community, what led him to establish

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39:33

Cardiovascular System Part 1: Blood

Patient Empowerment Program: A Rare Disease Podcast ·

2022/10/19

Focusing on the cardiovascular system, Stan starts a new lecture series on organs. No, not the state in the Pacific Northwest and definitely not the large piped musical keyboard you’d find in a church or a ballpark. We’re talking about the collection of tissues within the body that perform vital functions for life. Organs!Part 1 focuses on that warm red liquid inside of you, blood! Now blood is not technically an organ. But, according to Stan, it should be. Dr. Crooke covers how the blood cycle

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30:57

Coming Together for the Nano-rare Patient

Patient Empowerment Program: A Rare Disease Podcast ·

2022/10/05

When Stan Crooke, M.D., Ph.D., ran Ionis and John Maraganore, Ph.D., ran Alnylam, they were partners that turned into rivals — and not always friendly ones — as they persevered to pursue an entirely new therapeutic space; RNA-targeted drug discovery and development. Now, the pair of drug discovery titans have united once again in support of Dr. Crooke’s n-Lorem Foundation—working to provide experimental personalized medicines to the rarest of rare disease patients (nano-rare) using the antisense

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17:28

Antisense (How We Do It at n-Lorem)

Patient Empowerment Program: A Rare Disease Podcast ·

2022/09/21

We’d like to thank our sponsor, Argonaut Manufacturing Services. Argonaut provides contract manufacturing and sterile fill-finish services for biopharmaceutical, diagnostics, and life science organizations. Argonaut’s expertise in sterile fill-finish is the last step in the complex process of providing personalized antisense oligonucleotide (ASO) medicines to nano-rare patients.When considering if the technology he led the creation of could be a viable way to develop a drug for a single patient,

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43:09

Anna's Story of Hope and Help

Patient Empowerment Program: A Rare Disease Podcast ·

2022/08/31

In this episode, Stan talks with Sonja and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’s daughter, Anna. She is 17 years old and suffers an aggressive, fatal form of ALS. ALS is rare in teenagers, and this form of ALS is the worst of the worst. Sonja tells Anna’s story initial symptom onset, to diagnosis, and to Anna's response to ASO treatment. As a parent, Sonja reveals her full range of emotions during the duration of her daughter’s

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27:21

Why ASO Technology

Patient Empowerment Program: A Rare Disease Podcast ·

2022/08/17

What makes antisense oligonucleotides (ASOs) so special? Let’s first understand what an oligonucleotide is. An oligonucleotide is a short strand of synthetic DNA, or a nucleic-acid chain, usually consisting of up to approximately 20 nucleotides read from left to right. n-Lorem’s ASO technology is based on thirty years of innovation and investment to make ASOs drugs with optimal qualities. This technology is also uniquely beneficial for nano-rare patients due to its specificity and broad utility to

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29:40

Advocating for Rare Patients

Patient Empowerment Program: A Rare Disease Podcast ·

2022/08/03

In this episode, Stan speaks with Wendy Erler about her involvement in the treatment of patients with rare genetic disorders. From her start in the pharmaceutical industry, to her involvement with every major platform of drug development, to her position as vice president and head of patient advocacy at Alexion; Ms. Erler has always ensured that a patient's voice is heard.Learn more about Wendy Erler on twitter @wendy_erlerPlease like and subscribe so that we can reach more potential patients and

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34:33

Drug Discovery Platforms

Patient Empowerment Program: A Rare Disease Podcast ·

2022/07/20

There are three validated methods of discovering drugs – called platforms. Small molecule drugs (SMD), monoclonal antibodies (mAbs), and RNA-targeted drugs. SMDs are, you guessed it, very small! Remember that the size of chemicals is measured in units called Daltons, which is capitalized because it is named after the scientist who invented the term. The smallest chemical is a hydrogen ion, and it is 1 Dalton. Small molecule drugs are generally less than 500 Daltons. Because of this, they don’t carry

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