Motherhood is a journey filled with challenges, but every step is worth it for the love and joy it brings. Zoe Hummel joins the show in this special Mother’s Day episode to discuss her son Mostyn’s emotional story to diagnosis, and the unique challenges and triumphs of being a nano-rare mom. Order your Mother's Day card by May 6, 2024! All proceed support nano-rare patients. On This Episode We Discuss: How Zoe met her Husband, Mostyn’s father Zoe’s Violin origins and playing with Rod Stewart Mos...
May 01, 2024•50 min•Ep. 50
Research physicians and their associated institutions are monumental to the mission of the n-Lorem Foundation and are truly transformational to the lives of the nano-rare patients they treat. Olivia Kim-McManus, M.D., was one of four physician panelists at our first Colloquium who participated in "A Physician's Perspective on n-Lorem and Nano-rare". Hear Olivia's outlook on nano-rare patients and how the support of an institution like UCSD Rady Children’s Hospital makes all the difference. On Th...
Apr 17, 2024•49 min•Ep. 49
Genetics can be intricate, no doubt. With millions of mutations present in every human, one might wonder why not everyone is impacted by pathogenic diseases. The Patient Empowerment Program aims to assist you in grasping the fundamental concepts of various mutations and how they function, paving the way for you to enhance your understanding of genetics. Delve into the diverse array of disease-causing mutations, their characteristics, and explore which mutations could potentially be addressed thr...
Apr 03, 2024•26 min•Ep. 48
Each time one of the cells in your body divides to form a new cell, 3 BILLION genetic letters must be accurately duplicated. That’s a big number and mistakes DO happen in everyone. That’s right, if you’re reading this, you have a genetic mutation. On This Episode We Discuss: Your genetic alphabet – nucleotides How to think about DNA Replication Types of mutations What is an SNP and why you should care Indels The genetic code How genetic information is translated into a protein...
Mar 20, 2024•30 min•Ep. 47
What's one way to spread hope? By sharing your experiences and connecting with others who truly understand the everyday challenges nano-rare patients face. Shanna Tolbert was one of five nano-rare patient caregivers who participated in the panel, The Perilous Journey to Diagnosis and Treatment for Nano-rare Disease Patients. Listen to Shanna’s take on the importance of dialogue and relive a few patient stories as told by the parents who participated in this panel. On this episode, we discuss: 0:...
Mar 06, 2024•43 min•Ep. 46
For n-Lorem’s Chief Operating Officer, Sarah Glass, the mission of n-Lorem is personal. Her son Ethan was diagnosed with a nano-rare mutation. A geneticist by training, she joined n-Lorem to oversee and direct the organization’s efforts to provide hope and potential help to those who need it most. It's more than just a job for her; she's powered by her son and the entire nano-rare community. 1:20 Sarah’s background, early life, and scientific interests 7:20 Thinking about the patient experience ...
Feb 21, 2024•41 min•Ep. 45
Charissa Lipman joins n-Lorem founder and CEO, and host of the Patient Empowerment Program Podcast, Dr. Stan Crooke, in this question-and-answer episode to discuss additional questions asked during the 2023 Nano-rare Patient Colloquium. Charissa attended the inaugural Colloquium in October 2023 and brings the perspective of a patient family member, discussing her experiences and takeaways from the meeting. She is the mother of Ryker, a nano-rare n-Lorem patient with a CACNA1A genetic mutation. S...
Feb 07, 2024•46 min•Ep. 44
We're diving into the archives to resurface this top downloaded episode from last year. Dan Doctoroff joined Patient Empowerment Program in February 2023 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 out...
Jan 24, 2024•49 min•Ep. 43
For the return of the patient empowerment program podcast, we’re recapping the 2023 Nano-rare Patient Colloquium in this special “Best of” episode. For the Colloquium, we gathered the nano-rare community under one single roof for a full day of presentations and panels with experts, partners, supporters, and patients. So, we are bringing some of the top moments directly to your speakers. Discover the origins and aspirations of n-Lorem and Columbia University's Silence ALS initiative, visualize th...
Jan 10, 2024•39 min•Ep. 42
Nano-rare diseases strip power from families and this often leads to hopelessness, anger, and dread. However, channeling these intense emotions to carefully navigate risk/benefit decisions is within one’s control. n-Lorem is different than standard drug development settings. Every risk/benefit decision that n-Lorem makes is in the context of the individual patient’s symptoms, status, and needs. While we cannot promise benefit, we can promise that we will do our very best in the creation of an op...
Dec 13, 2023•35 min•Ep. 41
“How to Think About Risk” is arguably the most crucial topic ever discussed on the Patient Empowerment Program. Navigating decisions with substantial risk can be challenging, intricate, and even intimidating. The ability to detach from the intense emotions surrounding these choices is critical for making the best and most logical decisions. On This Episode We Discuss: Why nano-rare patients and their families need to think effectively about risk Risk vignettes: infectious diseases, automobile an...
Dec 06, 2023•43 min•Ep. 40
Jeff Carroll, Ph.D., inherited a gene that will eventually lead to symptoms of Huntington’s Disease. Alongside researching this debilitating disease as an Associate Professor of Neurology at the University of Washington, he’s a Scientific Advisor for n-Lorem and member of the Access to Treatment Committee (ATTC) that helps screen and assess submitted patient applications. On This Episode We Discuss: 2:45 Joining the Amy on a whim 4:30 Serving in Kosovo and Germany 6:00 Learning that his mother w...
Nov 29, 2023•39 min•Ep. 39
Chip Wilson, the creator of Lululemon and SOLVE FSHD, stands as a pioneer, business innovator, philanthropist, and a person affected by a rare disease. His passion for fitness led him to build a successful career in designing athletic wear, but a diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD), a degenerative muscle condition, altered his active lifestyle. On This Episode We Discuss: 0:46 Working on the Alaska Oil Pipeline as a 17-year-old 4:30 Saving money young and becoming an entre...
Nov 08, 2023•1 hr•Ep. 38
The Autonomic Nervous System automatically controls essential processes whether you’re awake or asleep. It’s the employee of the month. It gets the job done at any time of the day without you even having to ask it. It is our body’s autopilot system, controlling functions we often take for granted, like heart rate, digestion, pupil dilation, and even breathing! Life would be pretty hard on manual. On This Episode We Discuss: “I Sing The Body Electric” Hemo and chemo-electric machines The nervous ...
Oct 18, 2023•22 min•Ep. 37
Dr. Daniel Curran leads Takeda’s efforts to unlock innovation and deliver transformative medicines in a variety of rare-disease areas. Dan embraces learning from, collaborating with, and meeting members of the rare disease community in an effort to produce treatments that result in better health and a brighter future for rare. 2:02 How rare disease is defined at Takeda 4:10 Rare disease units at Takeda 5:23 How Takeda economically justifies half of their pipeline being rare disease drugs 9:06 Th...
Oct 04, 2023•30 min•Ep. 36
As the first ever Nano-rare Patient Colloquium is just around the corner, we are flipping the script. Spectrum News’ Brady Huggett interviews n-Lorem founder and CEO, Dr. Stan Crooke, and picks his mind to understand the inspiration behind the creation and want for this first of its kind nano-rare community event. Along with some background information on the highly anticipated Colloquium, Stan candidly conveys n-Lorem's hurdles and joys in its first four years of service.
Sep 20, 2023•36 min•Ep. 35
Liver disease is a broad term encompassing a range of conditions that affect the liver's structure and function. The liver is extremely important as it wears many different hats when it comes to your body functioning properly like controlling metabolism, storing and breaking down nutrients for use, detoxification and more! No wonder there are many diseases associated with liver disfunction including various genetic ones. Get ready to explore... you guessed it, various diseases of the liver! On t...
Aug 23, 2023•15 min•Ep. 34
The liver is a master chemist that sends copious signals to help control our appetite and breaks down fats to be stored and used as energy and to combat starvation. Next time you’re hangry, think of your liver kicking into overdrive. On This Episode We Discuss: Producing countless small chemical metabolites Controlling iron homeostasis The liver and the gut microbiome Combatting starvation and triggering appetite Managing glucose levels...
Aug 09, 2023•20 min•Ep. 33
The liver has many important and magical metabolic functions. It converts the nutrients in our diets into substances that the body can use and filters toxic substances out from the body. You won't believe the sheer metabolic mayhem that happens behind the scenes!
Jul 26, 2023•30 min•Ep. 32
Argonaut Manufacturing Services Inc. CEO, Wayne Woodard, joins the Patient Empowerment Program to detail how Argonaut bottles vials of hope for n-Lorem patients. Argonaut is n-Lorem's provider of Sterile Fill-Finish for an optimal ASO. This means that the lyophilized ASO powder, which was manufactured with good manufacturing practices (GMP), gets formulated and aliquoted into sterile vials. The final product is tested for various attributes to ensure maximal quality. Once assessed and approved, ...
Jul 12, 2023•30 min•Ep. 31
Gay Grossman is the mother of an n-Lorem patient and co-founder of ADCY5.org. She has been active in the rare disease space for 25+ years and continues to fight and advocate for patients every single day by connecting ADCY5 families, speaking at events, and informing the entire rare community. On Wednesday, Gay joins the Patient Empowerment Program podcast to discuss her daughter, Lilly, lessons that she has learned as a rare disease mom and advocate, the difficulty of gathering and sharing medi...
Jun 28, 2023•42 min•Ep. 30
Brace yourself for a deep dive into the various categories of lung disease. Obstructed airways and restricted blood flow pose significant risks to your body. So, learn preventive measures to safeguard your respiratory health. From pneumonia to the disruption of pulmonary blood flow, we'll explore the different culprits that block the flow of air and blood! On this episode we discuss: Obstruction of the airways due to smoking, cancer, infectious diseases, etc Obstruction of pulmonary blood flow O...
Jun 14, 2023•16 min•Ep. 29
Let's take a deep breath and fill our lungs with air. Ahh, doesn't that feel good? Our lungs are responsible for taking in oxygen from the air we breathe and distributing it to organs throughout our bodies and then ridding those organs of carbon dioxide. Get ready to dive into a fascinating episode on our lungs and how they protect us against particles, toxins, and infectious organisms in the air we breathe. The tinier the foreign particle, the more harmful they are! Let's explore the superhero-...
May 31, 2023•27 min•Ep. 28
May marks a year of podcasting for us, and we’re thankful for all those who have participated and the growing number of people who have listened to the podcasts. We are pleased with the overwhelming support for the podcast and the willingness of our partners and patients to share their stories. We launched our Patient Empowerment Program with the purpose of building a nano-rare community and establishing a forum in which the voices of patients, advocates, and experts can come together focused on...
May 17, 2023•46 min•Ep. 27
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 m...
May 03, 2023•32 min•Ep. 26
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 t...
Apr 19, 2023•51 min•Ep. 25
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 ...
Apr 05, 2023•22 min•Ep. 24
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 evi...
Mar 22, 2023•32 min•Ep. 23
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 ch...
Mar 08, 2023•25 min•Ep. 22
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’...
Feb 15, 2023•47 min•Ep. 21
