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 efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients (less than 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Dr. Stan Crooke, who founded IONIS Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. This podcast is produced by n-Lorem Foundation and hosted by Dr. Stanley T. Crooke, who is our Founder, CEO and Chairman. Our videographer is Jon Magnuson. Our producers are Kira Dineen, Jon Magnuson, Kim Butler, and Amy Williford. To learn more about n-Lorem, visit nlorem.org. Contact us at [email protected]
Fostering Collaboration to Fight ALS
31:53Manish 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 in their founding support of Silence ALS, an initiative that aligns Columbia University and n-Lorem to offer an integrated solution to identify, support and potentially treat nano-rare ALS patients. Target ALS hopes that one day, no patient, no matter how rare their form of ALS, is left behind. On This Episode We Discuss: Target ALS’ innovative model Accelerating ALS research through collaboration How a single nano-rare study can inform the ALS research landscapeThe importance of understanding the fundamental biology behind ALS Committing to fight all forms of ALSTarget AlS’ support for n-Lorem and Columbia University’s Silence ALS Initiative
The Energy of Life
25:02Regardless 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. The amount of chaos and disorder is measured in entropy. In this episode, the energy of life, entropy and how we convert, store and use energy is explored.Visit www.nlorem.org
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47:08Dan 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 inspiration to bring the Olympic Games to NYC 3 things he learned as a CEO Dealing with the reality of being diagnosed with ALS Shifting his perception of life How ALS has impacted his bloodline Scaling up Target ALS and fighting for the lives of every ALS patient
Cardiovascular System Part 4B: (The Spigot) Kidney Diseases
22:23If 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 kidney patients for treatment. Information about the application process and can be found at www.nlorem.org/access
Cardiovascular System Part 4A: (The Spigot) Kidney Functions
20:23There 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 kidney patients for treatment? Information about the application process and can be found at www.nlorem.org/access
Cardiovascular System Part 3: (The Pipes) Arteries and Veins
24:00Drip... 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 that control blood flow under unique conditions to supply our tissues with nutrients, remove waste, and keep us alive. In the event of a busted pipe, know that a plumber can’t help you. Call a doctor, because the pipes in your body are much more complex and interactive with the materials inside than the ones in your home.
CROs and Their Role for Nano-rare
27:59This episode is sponsored 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 moving the industry forward and advancing clinical research in healthcare’s most complex areas, like rare disease. Jamie Macdonald, CEO of Parexel, joins Stan to discuss the moment he realized his desire to make a difference, Parexel’s role in clinical trials and their partnership with n-Lorem aimed to serve patients.
Cardiovascular System Part 2B: (The Pump) Heart Diseases
22:25In 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.
Cardiovascular System Part 2A: (The Pump) Heart Anatomy
20:56In 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.
Shifting Mindsets to Expedite Rare Treatments
35:56This 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 the EveryLife Foundation, and the origins of Ultragenyx. Because of the debilitating and often quick progression of rare disease patients, Emil believes that regulatory agencies should modify the way they approach clinical trials for these individuals. Far too often, policies leave patients stuck waiting and hoping for treatment while their symptoms worsen, causing permanent damage. Do you have a question that you’d like to ask Stan Crooke? Stan will be taking questions directly from you and other podcast listeners and dedicating an entire episode towards answering your questions! To submit a question for the upcoming Q&A episode, email [email protected] with the subject line labeled “podcast question”. If you wish to be identified, mention your name in the email. If not, we will keep your submission anonymous. We can't wait to hear from you!