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Wait how do you spell that? is a rare disease podcast produced by Patient Worthy. We talk about issues affecting people rare and underdiagnosed conditions and interview advocates from across the community. We're definitely not doctors, and we can't give you medical advice. We're just here to chat and laugh and learn about issues that impact people living with diseases our doctors can't to spell.
 
I want to share information about living with and dealing with a chronic or invisible disease. This is made even more difficult if the disease is rare. I'll share my story and also invite some guests to share their viewpoints and experiences. There are hundreds of different types of diseases out there that are invisible and can the sufferer feel more alone. I'm hoping to help the patient, families and other support systems better communicate and empathize with each other. ***Disclaimer - Thi ...
 
If you or someone you love is affected by a rare disease, you likely have more questions than answers. That’s why we’re here. Rare Disease Connection, and our additional resources on RareDisease.com and YourDNA.com, brings together the people whose expertise can explain what you’re facing. From diagnosis, to prognosis, to treatment options, all the way to questions like “Who do I talk to? Where are the people who’ve been through this before?”. We have the answers, direct from the experts and ...
 
The Video Journal of Hematological Oncology (VJHemOnc) podcast covers the latest hematological oncology and hematology news from international experts – from leukemias, lymphomas, MDS and MPNs, to sickle cell, aplastic anemia and rare diseases. Made for healthcare professionals and researchers, we are a global, independent, open-access platform. Listen to the latest news, including cutting-edge trial updates, controversies and opinion. If you enjoy the podcast, please leave us a rating and r ...
 
Two Rare Mama Bears, a Cure CMD Podcast - where we discuss all things Congenital Muscular Dystrophy and Rare Disease...All subtypes, ages, abilities and topics with the intent to connect the CMD community. This podcast is about bringing together affected individuals, their family, researchers, clinicians, industry, and policy-makers to have conversations with the goal of moving the needle on the mission of Cure CMD. Hosts: Megan Meyer and Matty Manley.
 
Since 1989, Patient Services Incorporated has restored hope and health to chronic and rare disease patients. We truly admire the many perspectives we come across in our line of work. This inspired our new podcast, Rare Perspectives, where we go beyond the diagnosis and explore the heart of the experience. Our goal is to shed light on the paths that patients walk and highlight different perspectives. We hope their stories help to educate you about chronic illness.
 
Affecting less than 200,000 people in the US each year, sickle cell disease (SCD) remains a rare but painful and often misunderstood illness. Patients suffering from SCD often fly under the radar for critical early-stage diagnosis, which can lead to future complications and poor outcomes. This series seeks to bridge the gap in SCD education for healthcare providers on the frontlines of emergency medicine to improve patient care. Hear from experts in the field as they discuss common misconcep ...
 
I'm Aware That I'm Rare: the phaware® podcast is devoted to raising global pulmonary hypertension awareness with dynamic stories from PH patients, caregivers and medical professionals from around the world. Through this series of impactful, insightful and, most importantly, hopeful stories from members of the global pulmonary hypertension community, we hope to further the global #phaware conversation as well as to capture, engage and enable misdiagnosed and undiagnosed PH patients because ea ...
 
Welcome to Patient Paradox Podcast. My name is Mark Sleeper and I’m 30 yrs. old living with a chronic, life threatening disease called Cystic Fibrosis (or CF the easier name). I’m here to share the untold stories of some truly inspiring people who are battling or have battled life threatening health conditions. Along the way we will hear from brilliant doctors too.
 
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show series
 
Part 2. We are exploring back to school safety tips - even though some of the tips don’t necessarily apply to illnesses, they are good reminders on what we need to be aware of as our most precious resources begin another academic year. This episode will concentrate on keeping our kids healthy Contact: Email: Seetheinvisible2020@gmail.com Twitter: R…
 
Measurable residual disease (MRD) is emerging as an important prognostic marker in the management of multiple myeloma. Promising clinical evidence from trials, such as the MYELOMA XI trial, has linked MRD-negativity to improved outcomes in myeloma. Whilst research into MRD as a prognostic marker continues, MRD is further being explored as a tool to…
 
Autologous stem cell transplantation (ASCT) has been the standard of care patients with multiple myeloma for the past three decades; however, high-risk patients still have poor outcomes. Many bispecific antibody therapies are currently under investigation for the treatment of myeloma, such as blinatumomab, teclistamab, talquetamab and cevostamab, a…
 
Part 2. We are exploring back to school safety tips - even though some of the tips don’t necessarily apply to illnesses, they are good reminders on what we need to be aware of as our most precious resources begin another academic year. This episode will concentrate on transportation tips. Contact: Email: Seetheinvisible2020@gmail.com Twitter: Ronda…
 
Antibody therapies are rapidly transforming the treatment landscape of non-Hodgkin lymphoma (NHL). Since the development of rituximab as an anti-CD20 antibody, there has been an intense research interest in antibody-based therapeutic approaches. The use of antibody-drug conjugates, monoclonal antibodies, and bispecific antibodies are increasingly b…
 
A significant number of patients with acute myeloid leukemia (AML) are unable to withstand intensive chemotherapy regimens due to older age and the presence of comorbidities, creating an unmet medical need. Recent advances in the understanding of the genomics underlying AML have led to the development of therapies targeting FLT3, IDH1/2 and BCL-2. …
 
We are exploring back to school safety tips - even though some of the tips don’t necessarily apply to illnesses, they are good reminders on what we need to be aware of as our most precious resources begin another academic year. This episode will concentrate on keeping our kids healthy Contact: Email: Seetheinvisible2020@gmail.com Twitter: Ronda F J…
 
We are exploring back to school safety tips - even though some of the tips don’t necessarily apply to illnesses, they are good reminders on what we need to be aware of as our most precious resources begin another academic year. This episode will concentrate on transportation tips. Contact: Email: Seetheinvisible2020@gmail.com Twitter: Ronda F Jeffe…
 
Janus kinase (JAK) inhibitors are becoming an increasingly prominent class of drug in the treatment of myeloproliferative neoplasms (MPNs), in particular for the management of myelofibrosis symptoms. With the FDA approval of ruxolitinib in 2011 followed by the approval of fedratinib in 2019, JAK inhibitors are already established in the myelofibros…
 
Great progress has been made in the treatment of acute lymphocytic leukemia (ALL), primarily owing to the development of targeted therapies such as monoclonal antibodies and tyrosine kinase inhibitors. The use of targeted therapies has significantly improved survival outcomes for patients with ALL and has become a mainstay of frontline treatment. S…
 
This episode is completely about my experiences and thoughts as my children get ready to start the new school yeat with a new Variant, Delta,, of Covid 19 starting to surge. Contact: Email: Seetheinvisible2020@gmail.com Twitter: Ronda F Jefferson (@CInvisible2020) / Twitter Facebook: SeetheInvisible2020 | Facebook Instagram: Ronda Jefferson (@seeth…
 
Many clinical trials are currently investigating the potential benefits of interferons in the treatment of myeloproliferative neoplasms (MPNs), in particular for polycythemia vera, essential thrombocythemia and myelofibrosis. Interferon-alfa was originally explored for MPN treatment many years ago, however it’s frequent dosing and toxicity profile …
 
Real-world data gained through scientific studies is essential in the advancement of treatments for all hematologic malignancies. Historically, pharmaceutical companies have been restricted to randomized clinical trials as their only form of testing a treatment. However, due to technological advancement, there are now ways in which data from real-w…
 
Today I am reviewing some events that have happened to those with allergies and/or asthma while flying. This topic is very concerning to me as I have worried about what will happen if I need to fly, but an animal that I am allergic to is on the plane? And for clarity, I’m not referring to Service Animals but to pets. This topic can create hardships…
 
Over the past two decades the treatment landscape for non-Hodgkin lymphoma (NHL) has rapidly evolved, with the development of Bruton’s tyrosine kinase (BTK) inhibitors making a particularly significant impact and opening the door to the possibility of chemotherapy-free regimens. In 2013, ibrutinib became the first BTK inhibitor to receive FDA appro…
 
Research into the treatment of non-Hodgkin lymphoma (NHL) has made great progress within the last decade, with clinical trials such as the TRANSCEND-OUTREACH-007 and ZUMA-12 studies successfully advancing into later phases. Although there are currently various approaches to NHL treatment, primarily employing chemotherapy, steroid medication and all…
 
Immunotherapies are showing increasingly promising results for the treatment of patients with non-Hodgkin lymphoma (NHL) and, in particular, bispecific antibodies have demonstrated promising clinical results in heavily pre-treated patients. Several bispecific antibodies have entered the early stages of clinical trials since the approval of the CD19…
 
At present, four types of chimeric antigen receptor T-cell (CAR-T) therapy are FDA approved for specific types of lymphoma: for relapsed or refractory (R/R) mantle cell lymphoma, brexucabtagene autoleucel, for R/R follicular lymphoma, axicabtagene ciloleucel, and for some aggressive forms of non-Hodgkin lymphoma, axicabtagene ciloleucel, tisagenlec…
 
Asthma, Allergies and Travel. These words can strike fear in those who have to encounter all of these together. I will go over tips in this episode on how to make a trip safer. Note - I used the word "safer" because in some instances, even if you've taken appropriate steps, dander and other allergens can travel on other people's clothes. Contact: E…
 
Although classical Hodgkin lymphoma has a high cure and overall survival rate, the treatment of relapsed/refractory disease, treatment-related adverse events, quality of life and poor outcomes in older patients present ongoing unmet needs. Much research is currently focused on risk stratification, such as with positive emission tomography (PET) sca…
 
Hematopoietic stem cell transplantation (HSCT) is used as a treatment option for a wide variety of hematological disorders. Graft-versus-host disease (GvHD) is a systemic disorder that arises when the graft’s immune cells recognize the host as foreign and attack the recipient’s body cells. Its occurrence poses a major medical complication that limi…
 
Today I will explore how the ADA addresses Asthma and Allergies. Some may not realize that both Asthma and Allergies are considered disabilities and protected under the Abilities with Disabilities Act. Links to information regarding the ADA are in the description below. Source: Are Asthma and Allergies Disabilities? | AAFA.org ADA Information: ADA.…
 
The treatment of multiple myeloma is a rapidly evolving field with advances in the understanding of myeloma disease biology contributing to the development of novel targeted therapies. Recently, there has been a rapid increase in the number of chimeric antigen receptor (CAR) T-cell therapies being trialed for patients with multiple myeloma, and in …
 
In this episode Akshay Kumar and Richard Wang discuss how through innovative technology, decentralised clinical trials came to the limelight, facilitating remote access and ensuring continued operation of clinical trials. In addition, we discuss the future of decentralised clinical trials in a post-COVID world, its implications and demands within t…
 
In this roundtable discussion, chaired by Gilles Salles, MD, PhD, Memorial Sloan Kettering, New York, NY; Nirav N. Shah, MD, Medical College of Wisconsin, Brookfield, WI; Ulrich Jäger, MD, Medical University of Vienna, Vienna, Austria; Stephen Ansell, MD, PhD, Mayo Clinic, Rochester, MI, review the most exciting updates in lymphoma from the 2021 Eu…
 
In this roundtable discussion, chaired by Paolo Ghia, MD, PhD, Università Vita-Salute San Raffaele, Milan, Italy; Susan O’Brien, MD, University of California Irvine, Irvine, CA; Lydia Scarfò, MD, Università Vita-Salute San Raffaele, Milano, Italy and Othman Al-Sawaf, MD, University Hospital of Cologne, Cologne, Germany, review the most exciting upd…
 
Today I will review some of the information regarding when you're pregnant and have asthma. Source: https://www.aafa.org/asthma-during-pregnancy/ Disclaimer: I am in no way a medical, insurance or legal expert. If you have any questions in those areas, please be sure that you contact a specialist in that field. I will discuss topics or articles tha…
 
Myelodysplastic syndromes (MDS) are a group of myeloid clonal disorders caused by a defect in bone marrow, which hinders their ability to produce healthy and normal functioning red blood cells (RBCs). The heterogeneous nature of MDS means therapeutic approaches need to be specifically tailored to each subtype. Current treatment options for MDS incl…
 
Recent progress in the field of immunotherapy presents an exciting alternative to standard chemotherapy treatments for patients with non-Hodgkin lymphoma. However, at present, durable responses are only achieved in a minority of patients. Accumulating evidence suggests that the microenvironment plays a critical role in the regulation of immunothera…
 
The introduction of many novel drug classes and therapies has resulted in significant improvements in overall survival outcomes for patients with multiple myeloma over the past decade. Despite this, many patients become relapsed or refractory to currently available therapies and as such, novel agents and combination regimens remain key to improving…
 
The breakthrough discovery of upregulation of the JAK-STAT signaling pathway as the driving force behind myeloproliferative neoplasms (MPNs) has led to a broadened range of treatment options across the spectrum of MPNs. Myelofibrosis is one of the most common types of MPN and many clinical trials are exploring novel drugs and combinations for its t…
 
In part 3 of exploring Asthma, we look at what to look for in an infant or toddler who may have asthma. Disclaimer: I am in no way a medical, insurance or legal expert. If you have any questions in those areas, please be sure that you contact a specialist in that field. I will discuss topics or articles that I have found on publicly accessible mean…
 
In this episode, we discuss Leigh Syndrome, a rare mitochondrial disease, with Kasey Woleben and Sophia Zilber of the Cure Mito Foundation. We talk about the importance of patient registries and how rare disease patients and families band together to work for a better future. To learn more about Leigh Syndrome and the Cure Mito Foundation, visit th…
 
Over the past decade there has been a vast increase in the number of clinical trials taking place in the field of myeloproliferative neoplasms (MPNs), with many trials investigating the potential benefits of novel JAK inhibitors both as monotherapy and in combination with other drugs, such as Bcl-2, Bcl-xL or BET inhibitors. More recently, next-gen…
 
We continue to explore Asthma in this episode, as well as take more of a look at accessibility. There are different types of Asthma and the recognition of which type of a patient has is very important in treatment. The statistics on the impact of Asthma are staggering in loss of school, work, medical costs, and most importantly, human life. I know …
 
Treatment options for patients with myelodysplastic syndromes (MDS) remain limited, with the rapid advances seen in other hematological malignancies not replicated in this space. Aside from hematopoietic stem cell transplantation, current approaches are non-curative, instead aiming to delay disease progression and manage cytopenias. The development…
 
Over the last years, the chronic lymphocytic leukemia (CLL) field has seen significant growth in new drugs and new treatment combinations. One emergent strategy is fixed-duration therapy, which combines two or more agents for a set timeframe. Fixed-duration therapy is an appealing option for different reasons, including shorter treatment times and …
 
Acute myeloid leukemia (AML) is a clonal hematologic malignancy defined by genetic heterogeneity due to recurrent gene mutations. Precision medicine has gained importance in the treatment of AML, with important developments in gene mutation-targeted therapeutic agents transforming the treatment landscape. The role of measurable residual disease (MR…
 
The treatment and management of multiple myeloma has evolved drastically over the last ten years with the introduction of many novel drug classes and therapies, resulting in notable improvements in overall survival. However, many patients remain relapsed or refractory and thus clinical trials of novel agents and combinations are key to improving th…
 
We start to explore Asthma in this episode. There are different types of Asthma and the recognition of which type of a patient has is very important in treatment. The statistics on the impact of Asthma are staggering in loss of school, work, medical costs, and most importantly, human life. I know that I found many of these statistics a surprise and…
 
There are several forms of acute lymphoblastic leukemia (ALL), with B-cell ALL being the most common (~75% of cases). A range of treatments are available for ALL, including chemotherapy, radiation therapy, and stem cell transplantation. Chimeric antigen receptor T-cell (CAR-T) therapy is a novel cellular therapy that has demonstrated potential for …
 
In this episode, we speak with Kari Rosbeck of the TSC Alliance to discuss tuberous sclerosis syndrome, the importance of research and how the organization adapted during the COVID-19 pandemic.Learn more about tuberous sclerosis complex and the TSC Alliance here.Door Patient Worthy
 
In this episode, the P4A team take a deep dive into the Brazilian pharmaceutical market and discuss its potential for access to orphan drugs, cell and gene therapies. The overall Latin American pharma market is forecast to grow by 9% per year through to year 2028. However, major markets in this region such as Brazil have some major barriers to acce…
 
During the COVID-19 pandemic, myeloma support and advocacy groups were forced to adapt to a new way of working in order to continue helping patients with myeloma and improving their quality of life. The COVID-19 pandemic has changed the way that patients with myeloma interact with their doctors, with phone and video appointments filling in for face…
 
Over the past ten years, the approval of several new and effective therapies for multiple myeloma has significantly improved overall survival outcomes. The introduction of novel therapies has led to high levels of complete response, and many novel techniques are now being explored for the assessment of the depth of response, one of the most effecti…
 
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