Focusing on Innovation to Elevate Rare Disease Management
Clinical Advances to Improve Outcomes
While the number of patients affected by rare diseases may be low, recent data have shown that the clinical impact of these diseases on patient lives is immeasurable. Therapeutic advances are now focusing on more targeted, disease-specific approaches to provide true clinical innovation and elevate patient care. From hematology to endocrinology to dermatology and beyond, specialized approaches for rare diseases are without borders. Breakthrough treatments to more optimally manage rare diseases are coming: clinicians must be ready to provide patient-centered, multidisciplinary care so that their patients may benefit from these therapeutic advancements.
LEARNING MODULES
0.5 CME CREDIT • MAY 15, 2024
Multimodal Management of Inoperable CTEPH: Evolving Therapeutic Strategies
1 05-14-2015Nick H. Kim, MD
University of California San Diego
La Jolla, California, USA
In this module
- Importance of Multidisciplinary Collaboration in Managing Patients With CTEPH
- Analyzing the Treatment Landscape: Approaches to Treat Inoperable CTEPH
- The One-Two Punch: Evaluating the Efficacy for Multimodal Approaches in Inoperable CTEPH
- Honing In on Proactive Care to Enhance Safety on Multimodal Approaches in Inoperable CTEPH
- A Multipronged Approach to CTEPH Care: Strategies to Optimize Therapeutic Selection
0.5 CME CREDIT • MARCH 22, 2024
Easing the Diagnostic Journey for Both Children and Adults With Lennox-Gastaut Syndrome: Patient-Centered Strategies
2 03-21-2025M. Scott Perry, MD
Cook Children's Medical Center
Fort Worth, Texas, USA
Tracy Dixon-Salazar, PhD
San Diego, California, USA
In this module
- The Impact of Lennox-Gastaut Syndrome in Children and Adults
- Reaching a Differential Diagnosis of Lennox-Gastaut Syndrome in Children and Adults
- Suspecting Lennox-Gastaut Syndrome
- Treating Lennox-Gastaut Syndrome in Children and Adults
- Understanding the Impact of Lennox-Gastaut Syndrome: The Patient Perspective
0.5 CME CREDIT • FEBRUARY 15, 2024
Effective Communication Strategies for Improved Phenylketonuria Care
3 02-14-2025Stephanie Sacharow, MD
Medical Biochemical Geneticist
Boston, Massachusetts, USA
Brittany Holmes, MSN, APRN, FNP-BC
Boston, Massachusetts, USA
In this module
- Challenges Faced by Adults With Phenylketonuria
- Long-Term Management of Phenylketonuria: Dietary and Medical Approaches
- Importance of Effective Communication for Patients With Phenylketonuria
- Shared Decision-Making and Patient Empowerment
- Practical Strategies to Optimize the Management of Phenylketonuria
0.5 CME CREDIT • DECEMBER 15, 2023
Optimizing Care for Patients With Narcolepsy Type 1: Strategies to Personalize Treatment Plans With Novel Agents
4 14-12-2024Richard K. Bogan, MD, FCCP, FAASM
University of South Carolina School of Medicine
Columbia, South Carolina, USA
In this module
- Defining Narcolepsy Type 1 (NT1): The Importance of Addressing Both EDS and Cataplexy to Optimize Treatment Outcomes
- Assessing the Latest Efficacy Data for Novel Agents in NT1
- Reviewing the Latest Safety Data for Novel Agents in NT1
- Individualizing Treatment for NT1: Key Factors to Consider
- Patient Perspective: How Optimal Treatment for NT1 Enhances Quality of Life
0.5 CME CREDIT • NOVEMBER 15, 2023
Gene Therapy in Hemophilia A: Redefining the Treatment Paradigm
5 11-14-2024Wolfgang Miesbach, MD, PhD
University Hospital Frankfurt
Frankfurt, Germany
In this module
- Gene Therapy in Hemophilia A: What Does the Efficacy/Durability Data Say?
- Evaluating the Safety Data: How Safe Is Gene Therapy in Hemophilia A?
- Identifying Patients Eligible for Gene Therapy for the Treatment of Hemophilia A
- Implementation of Gene Therapy for the Treatment of Hemophilia A: Shared Decision-Making with Patients
- Short- and Long-Term Management of Hemophilia A: Monitoring Patients on Gene Therapy
0.5 CME CREDIT • SEPTEMBER 28, 2023
Itching for Relief: Exploring the Role of Novel Biologics in Prurigo Nodularis
6 09-22-2024Shawn Kwatra, MD
Department of Dermatology and Oncology
Johns Hopkins Itch Center
Baltimore, Maryland, USA
Victoria Rita Garcia-Albea, NP
Lahey Dermatology NP Training Program
Burlington, Massachusetts, USA
In this module
- Itching for Relief: Recognizing the Need for Treatment Intensification in Prurigo Nodularis
- Getting Under the Skin: The Rationale for Investigating Biologics in PN
- Finding a Path to Itch-Relief: Evaluating the Efficacy Data for Novel Biologics in PN
- On the Path to Safe Relief: Assessing the Safety Data for Novel Biologics in PN
- Optimizing the Use of Biologics to Alleviate Itch: A Look at the Clinical Considerations for Patients With PN
0.5 CME CREDIT • MARCH 9, 2023
Scratching the Itch: Biologic Breakthroughs in Prurigo Nodularis
7 03-08-2024Brian S. Kim, MD
Icahn School of Medicine at Mount Sinai
New York, NY, USA
0.5 CME CREDIT • DECEMBER 23, 2022
Blazing the Trail to Better Patient Outcomes: Erythroid Maturation Agents in β-thalassemia
8 12-22-2023Ashutosh Lal, MD
UCSF School of Medicine
University of California, San Francisco
UCSF Benioff Children's Hospital
Oakland, California, USA
Sujit Sheth, MD
Weill Cornell Medicine
New York Presbyterian-Weill Cornell
New York, New York, USA
0.5 CME CREDIT • DECEMBER 20, 2022
Diagnostic Imaging in Non-Cystic Fibrosis Bronchiectasis: The Radiologist's Role in Optimizing Care
9 12-19-2023Anne O'Donnell, MD
Georgetown University Medical Center
Washington, DC, USA
In this module
- Relevancy for the Radiologist: The Clinical Impact of Early Diagnosis
- Recognizing the Subtleties in NCFB: Opportunities to Advance Care
- Cardinal Symptoms and Red Flags: Improving Early Recognition of NCFB
- Treatment Advances in NCFB: Neutrophil Targeting to Address NCFB Pathophysiology
- Entering A New Treatment Paradigm: The Efficacy and Safety of Emerging Neutrophil-Targeted Therapies for NCFB
