Trusted knowledge base of resources & information, focused on treating & diagnosing .
Rare Disease Advisor is an online resource that provides health care professionals with comprehensive information on important principles of care in rare diseases as well as up-to-date clinical news on the diagnosis and treatment of these conditions. Website features include:
• Daily news
• Feature articles on key issues in rare disease
• Live conference coverage
• Expert opinion articles, includ
ing exclusive guest editorials from leading rare disease experts
• Concise drug monographs
• Medical slideshows
• Videos
• Continuing medical education (CME) activities
• And more...
Coverage of the following Rare Diseases:
Cardiology:
Long Chain Fatty Acid Oxidation Disorder (LCFAOD)
Pompe Disease
Gastroenterology:
Gastrointestinal Stromal Tumor (GIST)
Hematology:
Hemophilia
Sickle Cell Disease (SCD)
Systemic Mastocytosis (SM)
Hepatology:
Alagille Syndrome (ALGS)
Lysosomal Acid Lipase Deficiency (LAL-D)
Immunology:
Cold Agglutinin Disease (CAD or CAgD)
Hereditary Angioedema (HAE)
Myasthenia Gravis (MG)
Neurology:
Duchenne Muscular Dystrophy (DMD)
Multiple Sclerosis (MS)
Spinal Muscular Atrophy (SMA)
Hereditary Transthyretin Amyloidosis (hATTR)
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Oncology:
Cholangiocarcinoma (CCA)
Medullary Thyroid Carcinoma (MTC)
Diffuse Large B-Cell Lymphoma (DLBCL)
Pulmonology:
Alpha-1 Antitrypsin Deficiency (AATD)
Cystic Fibrosis (CF)
Idiopathic Pulmonary Fibrosis (IPF)
Pulmonary Arterial Hypertension (PAH)
“I learned that I had alpha-1 antitrypsin deficiency ( ) as a young adult, when I was about 25 years old. I eventually developed severe asthma-like symptoms. When I saw online that the life expectancy of a person with AATD was around 60 years old, I panicked.”
Read more from Alyssa Gloor as she highlights how life expectancy projections can often be a source of stress for members of the rare disease community and how they can often feel misleading 👉 https://brnw.ch/21wLshD
💡Discover Rare Disease Advisor's collection of quizzes!
📝 Test your knowledge and learn new insights while deepening your understanding of rare diseases.
Dive in and explore today: https://www.rarediseaseadvisor.com/clinicalqueries/
Test Your Knowledge About Digital Gait Outcomes in ARSACS Study Which of the following statements about the study on digital gait outcomes in autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), a differential diagnosis for Friedrich ataxia (FA), are correct? Select one of the options...
Researchers from the University of Texas reported the case of a patient with myasthenia gravis who had thymoma treatment and who developed anti-α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor encephalitis.
Read more: https://brnw.ch/21wLsc3
Researchers reported the case of a patient with MG who had thymoma treatment and who developed AMPA receptor encephalitis.
Over two-thirds of patients with neuromyelitis optica spectrum disorder ( ) experienced a “good” response after their initial attack of the disease when given either intravenous methylprednisolone or .
Read more: https://brnw.ch/21wLs3x
Over two-thirds of patients with NMOSD showed an improvement in clinical disability scores following treatment for initial attacks.
S*x could determine the age of onset of Huntington disease symptoms, according to a study recently published in Scientific Reports.
Read more: https://brnw.ch/21wLr7P
S*x could determine the age of onset of Huntington disease (HD) symptoms, according to a recently published study.
Frank Rivera was living in Florida with his wife in 2004. One day, while out for a run, he had trouble breathing and went to the hospital. “They took an x-ray and found masses in my lungs,” he recalled at the World Orphan Drugs Congress ( ).
Initially diagnosed with lung cancer after a biopsy, Rivera underwent treatment. However, another x-ray revealed lumps in his lungs, and this time, the biopsy diagnosed him with , a rare condition causing the immune system to overreact and form granulomas, especially in the lungs and lymph nodes. “I went through 4 years of chemo and radiation for a disease I never had,” Rivera said. “That really affected my mental health.”
Now the president of Stronger Than Sarcoidosis, Rivera began treatment for the rare disease. But in 2015, doctors ran out of treatment options. “I was in a lot of pain. I thought about committing su***de. I had it all planned out,” Rivera said.
People with rare disorders have a lower quality of life in all domains compared to the general population and those with common chronic diseases such as hypertension, arthritis, and asthma. They experience higher levels of anxiety, depression, and fatigue, and lower social engagement.
🤝Read more in the latest article from Manuela Callari, featuring tips on supporting the of the from Albert Freedman, PhD, the psychologist behind Rare Counseling: https://brnw.ch/21wLr3G
Prolonged uncertainty about the future is a common experience among rare disease patients and their families.
The first patient has been enrolled in the YT-YOLT-201-101 phase 1 trial set to evaluate YOLT-201, an in vivo , in patients with transthyretin amyloid polyneuropathy or .
Read more: https://brnw.ch/21wLqVi
The first patient has been enrolled in the phase 1 trial set to evaluate YOLT-201 in patients with ATTR-PN.
A study has confirmed that early with recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients with may reduce the risk of bleeding and delay the time to the first spontaneous bleed.
Read more: https://brnw.ch/21wLqM0
New data show that rFIXFc prophylaxis can reduce the risk of bleeding in previously untreated patients with hemophilia B.
Corticospinal tract pathology occurred earlier than expected in a YG8JR Friedreich ataxia mouse model, suggesting that corticospinal neuron damage is an earlier event than previously thought, according to a recently published study in BioRxiv & MedRxiv.
Read more: https://brnw.ch/21wLpQu
Corticospinal tract (CST) pathology occurred earlier than expected in a YG8JR Friederich ataxia (FA) mouse model, a study found.
is one of the current recommended treatments in the United States for patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis who exhibit acute kidney involvement. 📚 Kidney360
Read more: https://brnw.ch/21wLpDr
Plasmapheresis for decreasing the risk of ESKD and/or death in AAV remains to be elucidated, but the procedure is currently being recommended by the KDIGO.
Mirum’s phase 2b clinical trial studying for the treatment of primary biliary cholangitis showed positive preliminary results, according to a recently published press release.
Read more: https://brnw.ch/21wLoX9
A phase 2b clinical trial has shown positive preliminary results for volixibat intended for the treatment of PBC.
A report of five cases of newborns born to Rh-negative mothers that developed hemolytic disease of the fetus and newborn ( ) despite negative indirect antiglobulin testing and appropriate Rh immunoglobulin administration has been recently published in Indian Pediatrics.
Read more: https://brnw.ch/21wLout
In a recent report, five newborns born to Rh-negative mothers developed HFDN despite negative IAT and RHIG administration.
A study published in the journal Molecular Neurobiology determined crucial functional gut microbiota and potential associated with the pathogenesis of Huntington disease.
Read more: https://brnw.ch/21wLoq4
A new study determined crucial functional gut microbiota dysbiosis and potential biomarkers associated with HD pathogenesis.
A of a patient with undiagnosed systemic mastocytosis who experienced unexpected complications during has been recently published in Journal of Cardiothoracic & Vascular Anesthesia.
Read more on our newly launched SM Companion site➡️ https://brnw.ch/21wLoiJ
A recent report describes a patient with undiagnosed systemic mastocytosis who experienced unexpected complications during heart surgery.
A new single-center, randomized, open-label, crossover, phase 1 study is set to assess the relative bioavailability of HRS-5965 capsules and tablets for paroxysmal nocturnal hemoglobinuria in healthy participants.
Read more: https://brnw.ch/21wLoaj
A new study will compare the bioavailability of HRS-5965 capsules and tablets for paroxysmal nocturnal hemoglobinuria (PNH) in healthy volunteers.
❓What happens when a rare disease patient works in healthcare?
For Danielle Alstat, a primary biliary cholangitis (PBC) patient perspective contributor and respiratory therapist, it means creating a safe, supportive environment with plenty of understanding.
“My number one goal with each patient I work with is to ensure they feel safe and comfortable in my testing room. I am very consciously aware of others’ anxieties and fears, so I go out of my way to create a relaxing environment.”
Read more in Danielle’s latest column➡️ https://brnw.ch/21wLn9z
My journey with primary biliary cholangitis (PBC) has completely changed my way of thinking while working in healthcare.
Monitoring sleep patterns in the long term using wrist-worn wearable devices can be a potential symptom management strategy in patients with Huntington disease, according to a new study published in the Journal of Clinical Sleep Medicine.
Read more: https://brnw.ch/21wLn9a
Monitoring sleep patterns using wrist-worn wearable devices can be a potential symptom management stategy in patients with HD, a study found.
A literature review exploring the main challenges and controversies regarding hematopoietic transplantation for patients with has been recently published in the journal Frontiers .
Read more: https://brnw.ch/21wLn4M
A literature review exploring the main challenges regarding hematopoietic stem cell transplantation (HSCT) for patients with MF.
Patients with Lennox Gastaut syndrome appear to experience a significant clinical and economic burden, further illustrating the need for more therapeutic alternatives.
The study used closed-claims data to match over 9000 patients with LGS to healthy controls on a 1:1 basis, accounting for age and s*x. Read more:
Patients with (LGS) appear to experience a significant clinical and economic burden, illustrating the need for more therapeutic alternatives, a study found.
Myasthenia gravis should be considered in patients who develop unexplained pharyngeal dysphagia, according to a new study published in the journal The Laryngoscope.
Read more: https://www.rarediseaseadvisor.com/news/unexplained-pharyngeal-dysphagia-mg/
Mysathenia gravis (MG) should be considered in patients who develop unexplained pharyngeal dysphagia, a study found.
At the age of 15, a German girl named Anna began having trouble breathing and eating. She started losing weight and lost the ability to speak more than 2 or 3 words at a time. Ultimately, she needed a gastric tube to survive.
She was diagnosed with FUS P525L, a nano-rare form of amyotrophic lateral sclerosis ( ), the debilitating, fatal neurodegenerative disease usually seen in patients 40 to 70 years old.
Neil Shneider, MD, PhD, director of the Eleanor and Lou Gehrig ALS Center at Columbia University’s Department of , accepted Anna as a patient and treated her with , an experimental antisense oligonucleotide (A*O) that has helped her regain some elements of a normal life.
As Anna, now 19, showed the benefits of individualized treatment that targets the genetic cause of a disease, she was the proof-of-concept needed to establish the nonprofit N-Lorem Foundation.
The foundation, discovers and develops individualized A*Os for people with “nano-rare” diseases, defined as those affecting fewer than 30 patients worldwide. It commits to offer that treatment at no cost for life, as long as a patient’s physician says its benefits outweigh the risks.
Read more about the n-Lorem foundation here: https://www.rarediseaseadvisor.com/features/n-lorem-foundation-developing-treatments-worlds-rarest-diseases/
World Orphan Drugs Congress
Nano-rare diseases are defined as those affecting fewer than 30 patients worldwide.
💡 An international panel of physicians with expertise in myelofibrosis (MF) together with a diverse extended faculty developed new recommendations addressing the critical gaps in the management of MF.
There were 5 consensus themes encompassing 25 key clinical questions. These were defining the thresholds for anemia, and when to initiate or modify treatment, defining the threshold for thrombocytopenia and when to begin or adjust therapy, characterizing Janus kinase (JAK) inhibitor failure and what would call for switching therapy, when and how to evaluate prognosis in patients with MF, and unmet needs in MF clinical trials.
Read more: https://www.rarediseaseadvisor.com/news/new-recommendations-mf-management-published/
An international panel of physicians and a diverse extended faculty developed new recommendations for the management of MF, a study found.
Subacute liver injury was reported for the first time in infants less than 4 weeks of age with spinal muscular dystrophy who received , according to published in the journal Molecular Therapy – Methods & Clinical Development.
Read more:
Subacute liver injury occurred in 2 female infant cases after receiving gene therapy for SMA before 4 weeks of age, according to a case report.
A new study published in the Journal of Community Genetics has found several key psychosocial challenges to be common among Portuguese young adults with a genetic risk for transthyretin amyloid polyneuropathy (ATTR-PN).
Read more: https://www.rarediseaseadvisor.com/news/psychosocial-challenges-common-portuguese-young-adults-risk-attr-pn/
New research suggests that psychosocial challenges are common in Portuguese young adults at risk of transthyretin amyloid polyneuropathy (ATTR-PN).
The implementation of measurable residual disease assessment based on next generation sequencing could improve prognostic accuracy in acute lymphoblastic leukemia, as per a study in Cancer Cell International.
Read more: https://www.rarediseaseadvisor.com/news/next-generation-sequencing-mrd-could-improve-all-prognosis/
The implementation of MRD assessment based on next generation sequencing (NGS) could improve prognostic accuracy in a ALL, a study found.
Joseph Penrod, 21, from suburban Detroit, is a senior at Michigan State University pursuing a degree in journalism. Darrel Nicklow Jr., 26, from Arcadia, Florida, runs an online digital media business. Both men have Duchenne muscular dystrophy (DMD) and have attended numerous conferences focused on diagnosis, treatments, and clinical care.
However, these conferences often overlook daily life aspects such as mental health, careers, finances, relationships, and s*xuality.
A conversation between Joseph Penrod and his mother, Marissa Penrod led to the creation of "What Now? Possibilities in Adulthood with Duchenne," the first-ever virtual conference exclusively for older adolescents and adults with DMD. Sponsored by the nonprofit organization Team Joseph, the event will take place on July 17-18, 2024.
“We want to show kids and young men with Duchenne that there’s more to life than just drugs and going to doctor appointments and getting a new wheelchair,” Joseph said. “You can still have a job and make money. Just knowing I can work remotely from anywhere gives me hope.”
Learn more about the conference here: https://www.rarediseaseadvisor.com/features/virtual-conference-aims-help-young-men-duchenne-face-adulthood/
Sarepta Therapeutics NS Pharma Catalyst Pharmaceuticals, Inc. PTC Therapeutics Edgewise Therapeutics Dyne Therapeutics Pfizer CureDuchenne
With life expectancy growing among patients with DMD, young men with the disease are organizing an event to discuss living with it.
Taking on the challenge in South Africa wasn't just about proving himself - It was about showing the world what a person with Pompe disease can do.
"Through unwavering determination and indomitable spirit, I used encouragement from others to propel me forward, and I aimed to inspire countless people along the way. It took true grit and resilience, but in the end, I hope my journey to complete this event will serve as a powerful reminder that no obstacle is insurmountable if one dares to face it head-on."
Read more in Bruce Campbell's latest patient perspective: https://www.rarediseaseadvisor.com/patient-columns/inspiring-others-pompe-stand-ironman-strong/
A systematic review of 26 articles published in Clinical and Experimental Rheumatology found that current smokers with antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis are often younger, more often male, and have a unique disease profile.
Read more here: https://www.rarediseaseadvisor.com/news/smoking-influences-prevalence-and-prognosis-in-aav/
Current smokers with AAV are typically younger, predominantly male, and have different disease profiles compared with nonsmokers.
NTLA-2001, an investigational in vivo CRISPR-Cas9 genome editing therapy developed by Intellia Therapeutics, has shown potential for redosing, according to the first-ever clinical data presented at the Peripheral Nerve Society Annual Meeting.
Read more: https://www.rarediseaseadvisor.com/news/genome-editing-therapy-attr-ntla-2001-potential-redosing/
New data show that investigational in vivo CRISPR/Cas9 genome editing therapy for ATTR, NTLA-2001, has potential for redosing.
New evidence-based clinical practice guidelines developed to support primary care physicians treating individuals with were published recently in Haemophilia.
These guidelines include 82 recommendations divided into 13 categories, addressing organization of care, therapy for adults, and adaptations of therapy for pediatric care.
Read more: https://www.rarediseaseadvisor.com/news/evidence-based-guidelines-address-hemophilia-primary-care-setting/
A comprehensive literature review and expert consensus were used to develop the guidelines, ensuring their relevance and applicability to the primary care setting.
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Given the unique and complex needs of those with a rare disease, healthcare providers can play a crucial role in mitigating their mental health challenges. 📢Here are key strategies, Dr. Albert Freedman, the psychologist behind Rare Counseling shared in an interview with Rare Disease Advisor: 👂Listen actively: Taking the time to listen to patients and their families is invaluable. In healthcare systems where time is limited, this act alone can significantly reduce stress and anxiety. Patients need to feel heard and understood beyond their clinical symptoms. “Listening goes a long way,” Dr. Freedman said. “To listen and to get to know the person is a great gift in any doctor-patient relationship.” 🤝Build personal connections: Recognize that patients are more than their disease. Developing a relationship where patients feel known as individuals can foster trust and safety, which are crucial for their mental health. 🔍Acknowledge limitations and learn: Doctors should openly acknowledge the limits of their knowledge regarding rare diseases. “There are thousands of rare diseases, and we can’t expect every physician to know what they all are,” Dr. Freedman said. It’s critical to be honest with patients about what they know and don’t and show a willingness to learn more about their disease. This transparency helps build trust and reduces patient anxiety. “To be open to learning is really important because many of our rare disease patients are anxious when they find that they know more about their disease than the doctor does,” he said. 📚Seek and coordinate resources: When doctors cannot provide specific care due to a lack of knowledge about a rare disease, they should actively help patients find specialists or resources who can. Care coordination across specialties and healthcare providers can alleviate the burden on patients and their families. “Our healthcare systems are very siloed. Healthcare professionals should help coordina
Taking on the #Ironman challenge in South Africa wasn't just about proving himself - It was about showing the world what a person with Pompe disease can do. "Through unwavering determination and indomitable spirit, I used encouragement from others to propel me forward, and I aimed to inspire countless people along the way. It took true grit and resilience, but in the end, I hope my journey to complete this event will serve as a powerful reminder that no obstacle is insurmountable if one dares to face it head-on." Read more in Bruce Campbell's latest #PompeDisease patient perspective: https://www.rarediseaseadvisor.com/patient-columns/inspiring-others-pompe-stand-ironman-strong/ #PompeStrong
For those with Hemolytic Disease of the Fetus and Newborn (#HDFN), finding care isn't that simple. Often, specialists are few and far between, and in the state of Texas, there are only two. In a recent column, Taylor Jeans opens up about the challenges of an HDFN diagnosis and pleas for an increase in care. "In the entire state of Texas, there are only 2 doctors who are skilled enough to treat patients with HDFN. When I went to a doctor’s office in Dallas, the doctor was out on vacation and my child’s condition was critical. Hence, they had to call the only other specialized doctor in the state and transfer me to Houston." Read more from Taylor here➡️ https://brnw.ch/21wLfuu #RareDisease #obstetrics #FetalMedicine #MaternalFetalMedicine #Gynecology #Alloimmunization
Just as many individuals with rare diseases and disorders suffer from unmet medical needs, they also encounter significant informational gaps on the web. Patients and their families often search in vain for answers to their questions, finding instead content tailored for doctors, researchers, and investors — but not for patients and caregivers. Haymarket Media ’s Rare Disease Companion network of websites seeks to address this issue by providing patients and their families with news, educational materials, and trustworthy insights. Our goal is to deliver the answers and support you need in language that is accessible and understandable. Explore our Companion Sites here: HDFN Companion - https://www.hdfncompanion.com/ PBC Companion - https://www.pbccompanion.com/ SM Companion - https://www.smcompanion.com/ #RareDisease #rarediseasewarrior #RareDiseaseCommunity #careaboutrare #RareDiseases #rarediseasesupport
🩸Today is #WorldHemophiliaDay. Rare Disease Advisor joins the global community to raise awareness for those with #BleedingDisorders. 🔎To learn more about #Hemophilia visit https://brnw.ch/21wIUaL. #Hematology #HemoChat #HemophiliaCommunity #HemophiliaStrong #CareAboutRare #NotThatRare
Our friends at myCME have begun their Spring Flash Sale! 🌸🌈 Enjoy their PA Self-Assessments at 40% off with courses in internal medicine, family medicine, and urgent care medicine. Earn up to 40 credits (60 with with NCCPA bonus credits) today! https://brnw.ch/21wIcKr
Calling all healthcare heroes! Dive into RareDiseaseAdvisor.com, the web’s premier destination for clinicians dedicated to treating patients with rare diseases. 🦓💙 🔍 Explore a world of fresh, original, and authoritative content, designed to meet the unmet informational needs of healthcare professionals. Here's what makes us stand out: Daily Updates: Stay in the loop with our daily news coverage, featuring the latest in research, drug development, and rare disease advocacy. Exclusive Features: Explore impactful rare disease features and stories that provide a deeper understanding of the challenges and triumphs in the rare disease community. Expert Insights: Gain valuable knowledge through interviews with leading researchers and clinicians, plus comprehensive coverage of rare disease conferences. Resource Hub: Access expansive collections of informational pages on standards of care, therapies, and other HCP resources. On-the-Go Learning: Tune into insightful podcasts and multimedia offerings, tailored for the busy medical professional. At Rare Disease Advisor, we are on a mission to serve healthcare providers around the globe, bridging the gaps in news and knowledge for rare disease communities. Our goal is to be your go-to source for accurate, reliable information. 🚀 Join us in creating a community where information empowers, and together, let's make a difference in the world of rare diseases! #RareDisease #RareDiseaseAdvocate #RareDiseaseAwareness #Zebra #CareAboutRare #FindACure #BelieveInTheInvisible #HealthEquityInRare #RareDiseaseResearch #RareCommunity
November 1st is International Lennox-Gastaut Syndrome (LGS) Awareness Day 🌟 At Rare Disease Advisor, we're proud to support International LGS Awareness Day and join the global effort to raise awareness about this rare and challenging condition. Lennox-Gastaut Syndrome is a severe and rare form of epilepsy that typically manifests in early childhood. Understanding the basics of LGS is crucial for patients, caregivers, and healthcare professionals. 🔍 What is Lennox-Gastaut Syndrome (LGS)? LGS is characterized by multiple seizure types, intellectual and developmental disabilities, and often, behavioral disturbances. It can be a relentless battle for those affected and their families, making awareness and support essential. 🤝 How Can You Help? - Share this post to help us reach a wider audience. - Learn more about LGS, its symptoms, and the latest treatment options at https://brnw.ch/21wE3XE - Support organizations dedicated to improving the lives of LGS patients such as LGS Foundation, American Epilepsy Society, and CURE Epilepsy. #LGSAwareness #LennoxGastautSyndrome #EpilepsyAwarenessMonth #RareDisease #FindACure #CareAboutRare
This month, we stand united in shining a spotlight on Spinal Muscular Atrophy (SMA) - a rare genetic disorder affecting thousands around the world. 💙 🎗 What is Spinal Muscular Atrophy (SMA)? SMA is a neuromuscular disease that affects the motor neurons in the spinal cord, leading to progressive muscle weakness. It can affect individuals of all ages, from infants to adults, impacting their ability to walk, eat, and even breathe. 📚 Educate, Empower, and Advocate! Throughout this month, we'll be sharing vital information about SMA - its symptoms, treatments, and the latest research developments. Our aim is to empower patients, caregivers, and medical professionals with the knowledge they need to tackle SMA with strength and resilience. 🤝 Resources: At Rare Disease Advisor, we're committed to providing a safe space where individuals affected by SMA can discover valuable resources. We encourage you to visit https://fal.cn/3AnlZ to learn more. 🔹 Spread the Word: Help us amplify the voice of those living with SMA and raise awareness about this often overlooked condition. Share this post and use the hashtags #SMAwarenessMonth to show your support. Together, we can make a difference! #RareDisease #SMA #NeverGiveUp #RareDiseaseAwareness #CommunityMatters #RareDiseaseSupport #RareDiseaseAwarenessMonth #Hope #CureSMA
Today, as we celebrate the spirit of independence, let's remember the strength and resilience of those facing rare diseases. At Rare Disease Advisor, we stand in solidarity with all the incredible individuals and families navigating unique challenges every day. On this special day, we honor the brave souls who inspire us with their unwavering determination and extraordinary courage. We are dedicated to raising awareness, providing support, and driving research to improve the lives of those affected by rare diseases. Just as the fireworks light up the night sky, may the spark of hope ignite in every heart. Together, we can create a brighter future for everyone impacted by rare diseases. Wishing you a safe, joyful, and inclusive Independence Day!
Rare Disease Advisor would like to wish you a happy Memorial Day!
Today is World Cholangiocarcinoma Day. Cholangiocarcinoma (CCA) comprises a heterogeneous group of rare malignant tumors that originate from cells of the biliary tree. CCA is responsible for about 15% of all primary liver tumors and is the second most common primary malignancy of the liver, following hepatocellular carcinoma (HCC). It is also responsible for about 3% of gastrointestinal tumors. In hopes of helping create awareness, we would like to call attention to CCA and share our resource page for those who may be looking for more news, information, and patient perspectives: https://fal.cn/3vVak We would also like to call attention to the Cholangiocarcinoma Foundation and the Global Cholangiocarcinoma Alliance, two incredible organizations working to improve the lives of people with CCA. See how you can get involved with World Cholangiocarcinoma Day here: https://fal.cn/3vVai #WorldCCADay #Cholangiocarcinoma #CCA #BileDuctCancer #CureCCA #GreenForCCA
Happy Thanksgiving from Rare Disease Advisor and the rest of the Haymarket Medical Network!
Happy Fourth of July!
Marissa Penrod of Team Joseph talks about how it's okay to ask for help during the Community of Caregivers Panel at CureDuchenne #FUTURES #Duchenne #RareMoms #PatientAdvocacy #CaregiverAdvocacy
Senior Correspondent Larry Luxner, of Rare Disease Advisor, interviews Dr. Pradeep P.A. Mammen, Professor of Internal Medicine, Medical Director: Neuromuscular Cardiomyopathy Clinic, and Director of Transplant Research for the Advanced Heart Failure & Transplant Cardiology Program of UT Southwestern Medical Center. Dr. Mammen participated this weekend at CureDuchenne FUTURES 2022 Conference. Yesterday, Dr. Mammen spoke at the Track 2: Pillars of the Transition Journey and today he led a special breakout session on Female DMD Carriers. Here is part of the interview that will be coming out in the next few weeks in our Features section and Rare Disease podcast. Stay tuned. #LetsCureDuchenne #22FUTURES
The Exhibitor Showcase Mix and Mingle at CureDuchenne Futures 2022 Conference #LetsCureDuchenne
A moving moment from a mother discussing “not adding guilt” when it comes to approaches of treatment. CureDuchenne #22FUTURES
Happy New Year from all of us at Haymarket Medical Network!
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