Rare Disease Discussions
By Peter Ciszewski, CheckRare
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Category: Medicine
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Subscribers: 5
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Episodes: 135
Description
News and clinical perspective including CME programs focused on rare diseases. CheckRare focuses on rare and neglected diseases.
| Episode | Date |
|---|---|
|
Kidney Involvement in Lysosomal Disorders
|
Mar 05, 2024 |
|
HAE Treatment Advances: Highlights from ACAAI
|
Mar 03, 2024 |
|
Hereditary Angioedema: Current Treatment Options
|
Mar 03, 2024 |
|
Assessing, Monitoring, and Managing Respiratory Involvement in Lysosomal Disorders
|
Mar 03, 2024 |
|
Hereditary Angioedema: Current and Future Treatment Options
|
Jan 31, 2024 |
|
Alpha-Mannosidosis From a Mom’s Perspective, Featuring Rhonda Skipper
|
Dec 20, 2023 |
|
Diagnosing and Managing Alpha-Mannosidosis, Featuring Markey McNutt, MD, PhD
|
Dec 20, 2023 |
|
Signs and Symptoms of Alpha-Mannosidosis, Featuring Reid Sutton, MD
|
Dec 20, 2023 |
|
Alpha-Mannosidosis Overview, Featuring Laura Buch, MSPAS, PA-C
|
Dec 20, 2023 |
|
Myasthenia Gravis and the Complement System: Treatment Options
|
Dec 19, 2023 |
|
Myasthenia Gravis and the Complement System: Pathophysiology
|
Dec 19, 2023 |
|
Myasthenia Gravis and the Complement System
|
Dec 19, 2023 |
|
New Guidance To Treat Tumor-induced Osteomalacia (TIO)
|
Dec 01, 2023 |
|
New Guidance To Diagnose Tumor-induced Osteomalacia (TIO)
|
Dec 01, 2023 |
|
New Guidance To Diagnose and Treat Tumor-induced Osteomalacia (TIO)
|
Dec 01, 2023 |
|
Cushing's Disease Research Highlights: ENDO 2023
|
Oct 16, 2023 |
|
CME: Managing Cardiomyopathies in Lysosomal Disorders
|
Oct 05, 2023 |
|
Acromegaly Research Highlights: ENDO 2023
|
Oct 05, 2023 |
|
Thyroid Eye Disease: Overview, Diagnosis, and Treatment Options
|
Oct 03, 2023 |
|
Myasthenia Gravis Research Highlights: AAN 2023
|
Jul 15, 2023 |
|
Building and Maintaining a Multidisciplinary Team for Lysosomal Disorders
|
Jun 30, 2023 |
|
Myasthenia Gravis and the Need for Targeted Therapies
|
Jun 28, 2023 |
|
wAIHA Treatment-Options – Current and in Development (Chapter 3)
|
Jun 02, 2023 |
|
wAIHA Complications (Chapter 2)
|
Jun 02, 2023 |
|
wAIHA Pathophysiology and Diagnosis (Chapter 1)
|
Jun 02, 2023 |
|
Warm Autoimmune Hemolytic Anemia (wAIHA) - Full Program
|
Jun 02, 2023 |
|
CME: Fabry Disease Research Highlights
|
Apr 30, 2023 |
|
Gene Therapy to Treat Duchenne Muscular Dystrophy – Preliminary Clinical Trial Results
|
Mar 20, 2023 |
|
Growth Hormone Deficiency Research Highlights
|
Mar 13, 2023 |
|
Zero Relapses in Patients with NMOSD Given Ravulizumab
|
Mar 06, 2023 |
|
Hemophilia A Drug Given Priority Review Status
|
Feb 27, 2023 |
|
A Brief History of Newborn Screening
|
Feb 20, 2023 |
|
UT Southwestern Medical Center: a NORD Center of Excellence
|
Feb 13, 2023 |
|
Safety and Efficacy of PLN-74809 to Treat Idiopathic Pulmonary Fibrosis
|
Feb 06, 2023 |
|
New Report Estimates the Number of Rare Diseases is More Than 10,000
|
Jan 30, 2023 |
|
CME: Cushing’s Disease / Cushing’s Syndrome - Research Highlights
|
Jan 28, 2023 |
|
Positive Updated Results from MajesTEC-1 Study in Relapsed/Refractory Multiple Myeloma
|
Jan 23, 2023 |
|
CME: Pulmonary Arterial Hypertension (PAH) Research Highlights
|
Jan 15, 2023 |
|
Dr. Farber: The Impact of COVID-19 in the Diagnosis of Pulmonary Arterial Hypertension (PAH)
|
Jan 13, 2023 |
|
CME: The Immune System and Lysosomal Diseases
|
Dec 29, 2022 |
|
Growth Hormone Deficiency Research Highlights
|
Dec 14, 2022 |
|
PIK3CA-Related Overgrowth Syndrome (PROS)
|
Dec 14, 2022 |
|
Zero Relapses in Patients With NMOSD Given Ravulizumab
|
Nov 28, 2022 |
|
UT Southwestern Medical Center: a NORD Center of Excellence
|
Nov 16, 2022 |
|
Advances in Gene Therapy for Lysosomal Diseases
|
Nov 07, 2022 |
|
Advances in Gene Therapy for Lysosomal Diseases
|
Nov 02, 2022 |
|
Updates in Medical Management of Cushing’s Syndrome
|
Sep 27, 2022 |
|
Diagnosis and Comorbidities in Cushing’s Disease: New Consensus Summary Into Your Practice
|
Sep 27, 2022 |
|
Current and Emerging ERTs/SRTs
|
Sep 26, 2022 |
|
What is Dravet Syndrome?
|
Sep 07, 2022 |
|
What Is a Patient-Focused Drug Development (PFDD) Meeting?
|
Aug 30, 2022 |
|
Data From SHINE Study Shows Significant Breakthrough for Patients with Mantle Cell Lymphoma
|
Aug 12, 2022 |
|
What Is ENPP1 Deficiency?
|
Jul 27, 2022 |
|
PNH: Real-World Experience
|
Jul 12, 2022 |
|
Rare Diseases Cost Ten Times More Than Common Diseases
|
Jul 05, 2022 |
|
CME Webinar: Biomarkers in Lysosomal Storage Disorders
|
Jun 24, 2022 |
|
Hereditary Angioedema (HAE) Research Highlights: 2022 AAAAI Annual Meeting
|
Jun 07, 2022 |
|
An Interview With Dr. Raymond Wang About Mucopolysaccharidosis Type I (MPS I)
|
May 30, 2022 |
|
Positive Data from Regenxbio’s Gene Therapy Trials for MPS I and MPS II
|
May 16, 2022 |
|
What is Dystrophic Epidermolysis Bullosa?
|
Apr 22, 2022 |
|
The NORD Rare Disease Centers of Excellence
|
Apr 21, 2022 |
|
Data from Phase 3 Gene Therapy Trial in Hemophilia B Patients Very Encouraging
|
Apr 14, 2022 |
|
What Is Rett Syndrome?
|
Apr 06, 2022 |
|
Gene Therapy (RGX-111) to Treat Mucopolysaccharidosis Type I (MPS I)
|
Apr 01, 2022 |
|
Treatment Options for Myasthenia Gravis
|
Mar 23, 2022 |
|
Galactosemia Roundtable Discussion Overview
|
Mar 18, 2022 |
|
MPS II Research Highlights: WORLDSymposium 2022
|
Mar 18, 2022 |
|
Gaucher Disease Research Highlights: WORLDSymposium 2022
|
Mar 18, 2022 |
|
AADC Deficiency: Panel Discussion Overview
|
Mar 14, 2022 |
|
Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights from ASH 2021
|
Mar 08, 2022 |
|
Subasumstat + Rituximab Shown to Be Tolerable in Subsets of non-Hodgkin Lymphoma Patients
|
Mar 08, 2022 |
|
Dr Jerry Vockley Discusses Latest Phase 2 Data Assessing SYNB1618 To Treat Phenylketonuria (PKU)
|
Feb 28, 2022 |
|
Newborn Screening: Hemoglobinopathies and Newer Disorders on the RUSP
|
Feb 22, 2022 |
|
Newborn Screening: Metabolic Conditions
|
Feb 22, 2022 |
|
Newborn Screening: Talking to Parents
|
Feb 22, 2022 |
|
Newborn Screening: From RUSP to Reality
|
Feb 22, 2022 |
|
Pyruvate Kinase (PK) Deficiency Highlights from ASH 2021
|
Feb 18, 2022 |
|
Treatment Landscape for Relapsed and Refractory Multiple Myeloma
|
Feb 17, 2022 |
|
Teclistamab Monotherapy for Relapsed/Refractory Multiple Myeloma Patients
|
Feb 06, 2022 |
|
Lysosomal Storage Diseases: Central Symptoms and Comorbidities
|
Jan 27, 2022 |
|
Hereditary Angioedema (HAE) Highlights from ACAAI 2021 Annual Meeting
|
Jan 27, 2022 |
|
Positive Long-Term Data of Ibrutinib + Venetoclax as First-Line Treatment for Chronic Lymphocytic Leukemia (CLL)
|
Jan 24, 2022 |
|
Positive Safety and Efficacy Results in Rett Syndrome Study
|
Jan 12, 2022 |
|
Safety of the Bispecific Antibody, REGN5458, in Multiple Myeloma Patients
|
Jan 05, 2022 |
|
Sustained uMRD Demonstrated in Elderly CLL Patients Receiving Ibrutinib plus Venetoclax
|
Dec 23, 2021 |
|
Dr Anne Pariser Provides an Overview of NIH’s Office of Rare Diseases Research
|
Dec 19, 2021 |
|
New and Emerging Phenotypes in Lysosomal Storage Disorders
|
Dec 07, 2021 |
|
Retinoid Therapy for Congenital Ichthyosis Shows Promise
|
Nov 21, 2021 |
|
Overview of Congenital Ichthyosis
|
Nov 18, 2021 |
|
Milademetan Shows Promise as Treatment for Multiple Cancer Types
|
Oct 12, 2021 |
|
Overview of Juvenile Idiopathic Arthritis (JIA)
|
Oct 12, 2021 |
|
New Insights into Lysosomal Storage Diseases’ Pathophysiology is Changing Treatment
|
Jul 28, 2021 |
|
Overview of Sarcomas
|
Jul 22, 2021 |
|
Current Treatment Options for Bone and Soft Tissue Sarcomas
|
Jul 22, 2021 |
|
How the MMRF Is Advancing Multiple Myeloma Research
|
Jul 20, 2021 |
|
What is Krabbe Disease?
|
Jul 07, 2021 |
|
Gene Therapy Clinical Trial for Krabbe Disease is Recruiting Patients
|
Jul 07, 2021 |
|
TCGT: Overview, Symptoms, and the Need for a Multidisciplinary Approach
|
Jun 16, 2021 |
|
Acromegaly Highlights from ENDO 2021
|
Jun 11, 2021 |
|
Current and Emerging Treatments for Lysosomal Storage Diseases
|
May 05, 2021 |
|
Fabry Disease Research Highlights 2021
|
Apr 21, 2021 |
|
Prader-Willi Syndrome Overview
|
Apr 19, 2021 |
|
Investigational Drug Provides Improved Quality of Life for PNH Patients
|
Apr 15, 2021 |
|
What is Paroxysmal Nocturnal Hemoglobinuria?
|
Apr 15, 2021 |
|
Follicular Lymphoma and Marginal Zone Lymphoma
|
Apr 15, 2021 |
|
Phase 2 UNITY-NHL Study Regarding Umbralisib
|
Apr 15, 2021 |
|
FDA Approves Umbralisib for Marginal Zone Lymphoma, Follicular Lymphoma
|
Apr 15, 2021 |
|
Prader-Willi Syndrome: Overview and Potential Treatment
|
Apr 08, 2021 |
|
Rare Disease Clinical Trials: Study Designs and Common Concerns
|
Apr 08, 2021 |
|
Gaucher Disease Research Highlights
|
Mar 24, 2021 |
|
Eye on Neuromyelitis Optica Spectrum Disorder (NMOSD)
|
Feb 14, 2021 |
|
PAH Highlights from CHEST 2020
|
Feb 14, 2021 |
|
Fabry Disease Research Highlights
|
Jan 25, 2021 |
|
Gaucher Disease Research Highlights
|
Jan 25, 2021 |
|
Mucopolysaccharidoses (MPSs) Research Highlights
|
Jan 25, 2021 |
|
When to Suspect ATTR Amyloidosis
|
Jan 25, 2021 |
|
Diagnosing ATTR Amyloidosis
|
Jan 25, 2021 |
|
Spinal Muscular Atrophy and Genetic Counseling
|
Jan 25, 2021 |
|
Spinal Muscular Atrophy and Newborn Screening
|
Jan 25, 2021 |
|
Treating Spinal Muscular Atrophy
|
Jan 25, 2021 |
|
Diagnosing Spinal Muscular Atrophy
|
Jan 25, 2021 |
|
NETS Research Highlights
|
Jan 25, 2021 |
|
Rolling Submission for Cilta-cel to Treat Relapsed/Refractory Multiple Myeloma
|
Jan 17, 2021 |
|
Clinical Data Shows Promise for SCID Treatment
|
Jan 11, 2021 |
|
Gene Therapy Showing Promise in Treating XLRP
|
Oct 30, 2020 |
|
Narcolepsy Highlights from AAN 2020
|
Oct 26, 2020 |
|
NMOSD Highlights from AAN 2020
|
Oct 26, 2020 |
|
Newborn Screening and MPS I
|
Sep 11, 2020 |
|
Mucopolysaccharidosis I (MPS I) and Genetic Counseling
|
Sep 10, 2020 |
|
Treating Mucopolysaccharidosis I (MPS I)
|
Sep 10, 2020 |
|
Diagnosing Mucopolysaccharidosis I (MPS I)
|
Sep 10, 2020 |
|
Mucopolysaccharidosis I (MPS I) and Genetic Counseling
|
Sep 10, 2020 |
|
Tumor-induced Osteomalacia (TIO)
|
Jul 24, 2020 |
|
New Study to Treat Propionic Acidemia and Methylmalonic Acidemia
|
Jul 16, 2020 |
|
CheckRare: Diagnosing Gaucher Disease Before ERT
|
May 09, 2020 |