Patient Empowerment Program: A Rare Disease Podcast
Episodes
Realities of the Nano-rare: Episode 2 Oliver Glass
22 Apr 2026
Contributed by Lukas
A nano-rare diagnosis changes more than one life—it transforms an entire family. In this episode of Realities of the Nano-rare, n-Lorem CEO Stan C...
Realities of the Nano-rare: Episode 2 Oliver Glass
22 Apr 2026
Contributed by Lukas
A nano-rare diagnosis changes more than one life—it transforms an entire family. In this episode of Realities of the Nano-rare, n-Lorem CEO Stan Cro...
Realities of the Nano-rare: Episode 1 with Sarah Glass
08 Apr 2026
Contributed by Lukas
A nano rare diagnosis does not just affect one person. It reshapes an entire family. Realities of the Nano-rare takes you inside that experience. He...
Miracles of Science #2: Induced Pluripotent Stem Cells
25 Mar 2026
Contributed by Lukas
Welcome to part two of our series that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call them mir...
Where Emerging FDA Draft Guidance may Lead for Nano-rare Patients
04 Mar 2026
Contributed by Lukas
On This episode We Discuss: 2:30 – Andrew realized that finance plays a very large role in drug development and is an obstacle in for rare disease ...
Reflections from the n-Lorem Chairman | 2026
11 Feb 2026
Contributed by Lukas
In this video message, Chairman Stan Crooke reflects on the close of 2025, six years after founding n-Lorem. He shares his gratitude for the communi...
Miracles of Science: Genomics
04 Feb 2026
Contributed by Lukas
Today, we’re launching a NEW SERIES that pulls back the curtain on the scientific and medical breakthroughs that make n-Lorem possible. We call th...
Your Questions, Answered: 2026 Q&A
14 Jan 2026
Contributed by Lukas
Your questions, answered. n-Lorem CEO, Stan Crooke, answers questions directly from the nano-rare community in this special Q&A episode. How often...
Best of the 2025 Nano-rare Patient Colloquium
17 Dec 2025
Contributed by Lukas
In this special Best Of episode, we revisit some of the powerful moments from the 2025 Nano-Rare Patient Colloquium—an extraordinary gathering tha...
Natacha Gassenbach of Biogen: 2025 Hero of n-Lorem
03 Dec 2025
Contributed by Lukas
A conversation with Natacha Gassenbach, 2025 Hero of n-Lorem and Biogen leader. She shares Biogen’s decision to become a founding donor of n-Lorem, ...
Seeing with Grace: A Teen’s Journey with a Rare Vision-Affecting Disease
08 Oct 2025
Contributed by Lukas
Meet Grace Hoyt — a bright, witty high schooler with a passion for singing, art, and romantasy audiobooks. Grace also happens to live with a rare di...
Sneak Peak of the 2025 Nano-rare Patient Colloquium
24 Sep 2025
Contributed by Lukas
As the 2025 Nano-rare Patient Colloquium approaches, we welcome back Transmitter Features Editor Brady Huggett to guest-host this episode of the Patie...
A Conversation with the 2025 Heroes of n-Lorem
10 Sep 2025
Contributed by Lukas
The ‘Hero of n-Lorem’ Award is a recognition given to an individual volunteer or organization that has made substantial contributions to n-Lorem a...
Meet Rescue 7: Firefighters Serving Patients
27 Aug 2025
Contributed by Lukas
Rescue 7 is dedicated to serving the most vulnerable patient communities with care, compassion, and critical support. Inspired by the traditions of Fi...
Hope for Kinsley: Facing TUBB4A with Courage and Care
06 Aug 2025
Contributed by Lukas
Sarah and Ryan Marta, proud parents of three girls from Central Texas, began noticing that their middle daughter, Kinsley, was slow to reach mobility ...
New Insights into Genetically Caused Developmental Delays
23 Jul 2025
Contributed by Lukas
Emerging observations from our work at n-Lorem suggest that not all developmental delays are permanent. In the absence of structural damage—such as ...
Research to Advance ASO Technology at n-Lorem
09 Jul 2025
Contributed by Lukas
At n-Lorem, every investment decision carries real weight by directly affecting the nano-rare patients we serve. We think very deeply about each decis...
Commitment to Safety First: The DSMB
25 Jun 2025
Contributed by Lukas
The Data Safety Monitoring Board (DSMB) is composed of distinguished, independent experts in antisense oligonucleotide (ASO) technology, clinical tria...
Connor Gooley's Story: A First for TUBB4A Treatment
11 Jun 2025
Contributed by Lukas
Connor Gooley is the first patient ever treated with an ASO for TUBB4A-Related Leukodystrophy, a condition that severely disrupts his nervous system, ...
What Are iPSCs—and Why Do They Matter?
28 May 2025
Contributed by Lukas
Induced pluripotent stem cells (iPSCs) are a groundbreaking, and mind-blowing, scientific advancement—one of many that help make it possible for n-L...
Biological Systems & Chemical Networks
14 May 2025
Contributed by Lukas
What Are Chemicals? | How Drugs Work in the Body | Understanding Homeostasis We’re all made of chemicals—but what exactly is a chemical? In this v...
Why ASO Technology?
30 Apr 2025
Contributed by Lukas
What makes antisense oligonucleotides (ASOs) so special? Let’s first understand what an oligonucleotide is. An oligonucleotide is a short strand of...
Connor Dalby's Story: n-Lorem Patient #001 with Kelley Dalby and Dr. Olivia Kim-McManus
16 Apr 2025
Contributed by Lukas
Connor Dalby was born with an SCN2A ion channel mutation causing severe epilepsy and eventually leading to hospice care at an early age. It was a c...
Our Mission is Personal with Sarah Glass
02 Apr 2025
Contributed by Lukas
For n-Lorem's Chief Operating Officer, Sarah Glass, the mission of n-Lorem is deeply personal. Her son Ethan was diagnosed with a nano-rare mutation, ...
How Cells Phone a Friend: Local Communication
19 Mar 2025
Contributed by Lukas
Our bodies are like bustling cities of cells, always chatting and working together. They don’t just send long-distance messages to organs—they als...
How Cells Phone a Friend: Long Distance Communication
05 Mar 2025
Contributed by Lukas
How do cells communicate with each other? With trillions of cells in the human body, seamless collaboration is essential within this intricate cellula...
Rosie’s Story: A Nano-rare Treatment Pioneer
19 Feb 2025
Contributed by Lukas
Rosie was the first of three HNRNPH2 patients to receive treatment with an n-Lorem ASO, helping to pave the way for others. Her parents, Nicole and Bo...
Sloane's Story: A Shared Medicine
05 Feb 2025
Contributed by Lukas
Sloane was born with a KIF1A mutation and is a special case at n-Lorem—she was the first patient to receive a medicine originally designed for someo...
5-years of n-Lorem: What Have We Learned?
22 Jan 2025
Contributed by Lukas
Five years of creating medicines, instilling hope, and overcoming steep challenges—all with the singular goal of improving the lives of nano-rare di...
I Sing the Body Electric
08 Jan 2025
Contributed by Lukas
Walt Whitman’s 1855 poem, I Sing the Body Electric, is a celebration of the human body and its intrinsic connection to the universe. During that era...
Q&A - 2024 Nano-rare Patient Colloquium Recap
18 Dec 2024
Contributed by Lukas
Join Stan Crooke, n-Lorem founder, CEO, and host of the Patient Empowerment Program Podcast, alongside Amy Williford, Sr. Director of Communicatio...
Best of the 2024 Nano-rare Patient Colloquium
04 Dec 2024
Contributed by Lukas
This year’s Colloquium spanned two perfect autumn days in beautiful Cambridge, Massachusetts where hundreds of nano-rare community members from arou...
Susannah's Progress: Two Years of ASO Treatment with Dr. Jennifer Bain and Luke Rosen
16 Oct 2024
Contributed by Lukas
Two years of treatment—two years of strength, courage, and blazing a trail for other nano-rare patients! 🎉 Susannah's story was the first shared ...
Sneak Peek of the 2024 Nano-rare Patient Colloquium
02 Oct 2024
Contributed by Lukas
Take a glimpse behind the curtain as we offer you a sneak peek of the 2024 Nano-rare Patient Colloquium. We’re thrilled to welcome back longtime bio...
The Building Blocks of What's Possible with David Butler, Ph.D.
18 Sep 2024
Contributed by Lukas
Hongene Biotech is a producer of RNA building blocks – the first step for what’s possible in RNA drug-discovery and development. David Butler, Ph....
Knowledge is Power: Part 2
03 Sep 2024
Contributed by Lukas
The concluding episode of the Knowledge is Power series. The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patient...
Knowledge is Power: Part 1
21 Aug 2024
Contributed by Lukas
The knowledge we are gaining at n-Lorem has even more value than benefiting nano-rare patients and their families. These insights will enable scientis...
Adopting a Rare Son with Paul Compton
07 Aug 2024
Contributed by Lukas
Paul Compton and his wife Andrea chose to start a family through adoption. Unknown to anyone, one of their adopted boys, Taeson, was the inheritor of ...
Ion Channel Mutations: The Complexities of Treatment
24 Jul 2024
Contributed by Lukas
Mutations affecting ion channels are the most common cause of 'applications for treatment' submitted to n-Lorem. These channels regulate the passage o...
Why We Dose Intrathecally
10 Jul 2024
Contributed by Lukas
The affected organs of our patients determine the route of administration for an ASO, as certain routes can better target specific organs and offer th...
Behind the ASO Design and Discovery with Konstantina Skourti-Stathaki, Ph.D.
26 Jun 2024
Contributed by Lukas
Konstantina Skourti-Stathaki leads the n-Lorem research team behind the design and discovery of ASOs. Nadina, as we at n-Lorem call her, has a passion...
Best of the 2023 Nano-rare Patient Colloquium
12 Jun 2024
Contributed by Lukas
We’re recapping the 2023 Nano-rare Patient Colloquium in this special “Best of” episode. For the Colloquium, we gathered the nano-rare community...
The Hurdles of Insuring Patients with Nano-rare Mutations with Alan Lotvin, M.D.
29 May 2024
Contributed by Lukas
What's preventing insurers from covering the specialized care of patients with nano-rare mutations, thereby expanding access to available treatment op...
Anna’s Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider
15 May 2024
Contributed by Lukas
Stan Crooke speaks with Sonja Kampfer and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’s...
Mostyn’s Story: Mother’s Day Special with Zoe Hummel
01 May 2024
Contributed by Lukas
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 M...
Physicians and Institutions Transform the Lives of Nano-rare Patients with Olivia Kim-McManus, M.D.
17 Apr 2024
Contributed by Lukas
Research physicians and their associated institutions are monumental to the mission of the n-Lorem Foundation and are truly transformational to the li...
Advanced Genetics Part 2
03 Apr 2024
Contributed by Lukas
Genetics can be intricate, no doubt. With millions of mutations present in every human, one might wonder why not everyone is impacted by pathogenic di...
Advanced Genetics Part 1
20 Mar 2024
Contributed by Lukas
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...
Sharing Hope Through Storytelling & Connection with Shanna Tolbert
06 Mar 2024
Contributed by Lukas
What's one way to spread hope? By sharing your experiences and connecting with others who truly understand the everyday challenges nano-rare patients ...
Our Mission is Personal with Sarah Glass
21 Feb 2024
Contributed by Lukas
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 gen...
Q&A #2 with Patient Families
07 Feb 2024
Contributed by Lukas
Charissa Lipman joins n-Lorem founder and CEO, and host of the Patient Empowerment Program Podcast, Dr. Stan Crooke, in this question-and-answer episo...
Everybody Lives with Dan Doctoroff
24 Jan 2024
Contributed by Lukas
We're diving into the archives to resurface this top downloaded episode from last year. Dan Doctoroff joined Patient Empowerment Program in February 2...
Best of the 2023 Nano-rare Patient Colloquium
10 Jan 2024
Contributed by Lukas
For the return of the patient empowerment program podcast, we’re recapping the 2023 Nano-rare Patient Colloquium in this special “Best of” episo...
How to Think About ”Risk” Part 2
13 Dec 2023
Contributed by Lukas
Nano-rare diseases strip power from families and this often leads to hopelessness, anger, and dread. However, channeling these intense emotions to car...
How to Think About ”Risk” Part 1
06 Dec 2023
Contributed by Lukas
“How to Think About Risk” is arguably the most crucial topic ever discussed on the Patient Empowerment Program. Navigating decisions with substant...
Inheriting Huntington’s Disease and Aspiring to Prolong Lives with Jeff Carroll
29 Nov 2023
Contributed by Lukas
Jeff Carroll, Ph.D., inherited a gene that will eventually lead to symptoms of Huntington’s Disease. Alongside researching this debilitating disease...
Living a Longer, Healthier, and More Fun Life with Chip Wilson
08 Nov 2023
Contributed by Lukas
Chip Wilson, the creator of Lululemon and SOLVE FSHD, stands as a pioneer, business innovator, philanthropist, and a person affected by a rare disease...
Autopilot: The Autonomic Nervous System
18 Oct 2023
Contributed by Lukas
The Autonomic Nervous System automatically controls essential processes whether you’re awake or asleep. It’s the employee of the month. It gets th...
Better Health, Brighter Future for Rare with Dan Curran
04 Oct 2023
Contributed by Lukas
Dr. Daniel Curran leads Takeda’s efforts to unlock innovation and deliver transformative medicines in a variety of rare-disease areas. Dan embraces ...
Sneak Peek of the 2023 Nano-rare Patient Colloquium
20 Sep 2023
Contributed by Lukas
As the first ever Nano-rare Patient Colloquium is just around the corner, we are flipping the script. Spectrum News’ Brady Huggett interviews n-Lore...
Liver: Diseases
23 Aug 2023
Contributed by Lukas
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 i...
Liver: The Master Chemist
09 Aug 2023
Contributed by Lukas
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 comb...
Liver: Metabolic Magic
26 Jul 2023
Contributed by Lukas
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...
Bottling Hope
12 Jul 2023
Contributed by Lukas
Argonaut Manufacturing Services Inc. CEO, Wayne Woodard, joins the Patient Empowerment Program to detail how Argonaut bottles vials of hope for n-Lore...
Pursuing Diagnoses and Sequencing
28 Jun 2023
Contributed by Lukas
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 continue...
Lungs: Heavy Breathing — Diseases and Obstructions
14 Jun 2023
Contributed by Lukas
Brace yourself for a deep dive into the various categories of lung disease. Obstructed airways and restricted blood flow pose significant risks to you...
Lungs: Defending Airways
31 May 2023
Contributed by Lukas
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 brea...
One Year Anniversary: It Takes a Rare Community
17 May 2023
Contributed by Lukas
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 ...
Coming Together for the Nano-rare Patient
03 May 2023
Contributed by Lukas
This episode is proudly sponsored by our partner, Alnylam Pharmaceuticals, the leading RNAi therapeutics company. When Stan Crooke ran Ionis and John ...
Q&A #1 with Patient Families
19 Apr 2023
Contributed by Lukas
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 Sh...
Bits and Bytes Help Streamline Bench to Bedside
05 Apr 2023
Contributed by Lukas
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 Pati...
Fostering Collaboration to Fight ALS
22 Mar 2023
Contributed by Lukas
Manish Raisinghani joins the Patient Empowerment Program to discuss how Target ALS fosters collaboration between academia and industry to research and...
The Energy of Life
08 Mar 2023
Contributed by Lukas
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 ...
Everybody Lives
15 Feb 2023
Contributed by Lukas
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 ...
Cardiovascular System Part 4B: (The Spigot) Kidney Diseases
01 Feb 2023
Contributed by Lukas
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...
Cardiovascular System Part 4A: (The Spigot) Kidney Functions
18 Jan 2023
Contributed by Lukas
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...
Cardiovascular System Part 3: (The Pipes) Arteries and Veins
04 Jan 2023
Contributed by Lukas
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 o...
CROs and Their Role for Nano-rare
14 Dec 2022
Contributed by Lukas
This episode is sponsored by our partner, Parexel, one of the world’s largest clinical research organizations (CROs) providing the full range of Pha...
Cardiovascular System Part 2B: (The Pump) Heart Diseases
30 Nov 2022
Contributed by Lukas
In this episode, Stan continues his lecture series on organs, concluding his lesson on the pump of the cardiovascular system, the Heart. Organs are in...
Cardiovascular System Part 2A: (The Pump) Heart Anatomy
16 Nov 2022
Contributed by Lukas
In this episode, Stan continues his lecture series on organs, focusing on the pump of the cardiovascular system – the Heart. Organs are individual t...
Shifting Mindsets to Expedite Rare Treatments
02 Nov 2022
Contributed by Lukas
This episode is sponsored by our partner, Ultragenyx, a biopharmaceutical company focused on the development and commercialization of novel therapies ...
Cardiovascular System Part 1: Blood
19 Oct 2022
Contributed by Lukas
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 l...
Coming Together for the Nano-rare Patient
05 Oct 2022
Contributed by Lukas
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 friend...
Antisense (How We Do It at n-Lorem)
21 Sep 2022
Contributed by Lukas
We’d like to thank our sponsor, Argonaut Manufacturing Services. Argonaut provides contract manufacturing and sterile fill-finish services for bioph...
Anna’s Story of Hope and Help
31 Aug 2022
Contributed by Lukas
In this episode, Stan talks with Sonja and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’...
Why ASO Technology
17 Aug 2022
Contributed by Lukas
What makes antisense oligonucleotides (ASOs) so special? Let’s first understand what an oligonucleotide is. An oligonucleotide is a short strand of ...
Advocating for Rare Patients
03 Aug 2022
Contributed by Lukas
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 ph...
Drug Discovery Platforms
20 Jul 2022
Contributed by Lukas
There are three validated methods of discovering drugs – called platforms. Small molecule drugs (SMD), monoclonal antibodies (mAbs), and RNA-targete...
Clinical Trials for One Patient
06 Jul 2022
Contributed by Lukas
In this episode, Stan talks to Dr. Joe Gleeson about the role of genetics in neurology and pediatric patients with neurological conditions. Early in ...
How Drugs are Used
22 Jun 2022
Contributed by Lukas
Drugs are chemicals. They are like all other chemicals except that humans make a value judgement that a particular chemical (drug) will effect a desir...
A Diagnostic Odyssey: The UDN and n-Lorem’s Roles
08 Jun 2022
Contributed by Lukas
Dr. Sessions Cole shares his career being a neonatal pulmonologist and his involvement in the undiagnosed diseases network, the UDN. Dr. Cole estimat...
Biological Systems & Chemical Networks
25 May 2022
Contributed by Lukas
We are all chemical engines. So, what exactly is a chemical? Chemicals are anything that occupies space in the universe and exist in either a solid, l...
What is n-Lorem?
25 May 2022
Contributed by Lukas
n-Lorem provides an opportunity for a better life, renewed hope and brighter dreams, one patient, one family at a time. Our mission is to provide pers...
Susannah’s Story with Dr. Wendy Chung and Luke Rosen
25 May 2022
Contributed by Lukas
Dr. Stan Crooke talks to Luke Rosen and Wendy Chung about Luke’s seven-year old daughter, Susannah. Susannah has a genetic mutation that is progres...
Patient Empowerment Program
16 May 2022
Contributed by Lukas
We are excited to announce n-Lorem’s brand new podcast, the "Patient Empowerment Program". There are podcasts about genetics and rare diseases, but ...