14:02

The GRIN2B Foundation

17:04

Results from Clinical Trial Testing Investigational Drug for GRIN-Related Disorders

12:59

Genetic Associations With ALS Reversals

14:58

Investigational Therapy for Dermatomyositis

06:29

FDA Approves Treatment for Chronic Graft Versus Host Disease

13:04

Global Cancer Burden Among Men: Disparities and Projections

16:35

Advancements in the Treatment of Myasthenia Gravis

14:10

Addressing the Challenges of Hypercortisolism in Cushing’s Syndrome

10:04

Accelerated Approval for IgA Nephropathy Treatment

12:03

Pegcetacoplan in Patients With C3G and Primary IC-MPGN

15:44

Treatment Effect on Patient Pain and Quality of Life in Neuromyelitis Optica

08:46

Navigating Diagnosis and Treatment for Primary Biliary Cholangitis: The Role of the Patient Voice

08:11

Long-Term Data of Trofinetide in Patients With Rett Syndrome

10:30

Long-Term Data of Trofinetide in Patients With Rett Syndrome

14:21

Expanded Label of Treatment for Primary Immunodeficiencies

06:19

Long-Term Data Testing Treatment in Patients With Thyroid Eye Disease

55:52

Patient Perspective: Journey to a Myasthenia Gravis Diagnosis

13:42

Gene Therapy Long-Term Data for Retinal Dystrophy

19:51

CTCL: Shortening the Diagnostic Journey and Starting Treatment Early (Full Interview)

03:48

CTCL Overview (Chapter 1)

04:52

Treatment Plans for CTCL (Chapter 2)

02:42

CTCL: The Role of the Dermatologist in Caring for Patients (Chapter 3)

05:57

Global Care Collaborative Pioneers Consensus for Improving Diagnosis and Care in CTCL (Chapter 4)

13:16

Breakthrough Therapy for CIDP Treatment

13:17

Phase 2 Trial Evaluating LUM-201 for Treatment of Growth Hormone Deficiency

12:53

CTCL: The Role of Dermatologists in Diagnosing and Caring for Patients (Full Interview)

03:52

Challenges of Diagnosing CTCL (Chapter 1)

02:52

Global Care Collaborative Pioneers Consensus for Improving Diagnosis and Care in CTCL (Chapter 4)

04:07

Ensuring the Patient Is at the Center of the CTCL Care Team (Chapter 3)

01:49

The Role of the Dermatologist in Diagnosing and Caring for CTCL Patients (Chapter 2)

09:55

Clinical Trial: Sparsentan for Treatment of IgA Nephropathy

05:29

Living With Hypoparathyroidism and the HypoPARAthyroidism Association

06:53

Challenges of Hypoparathyroidism

05:54

Clinical Trial Evaluating Investigational AVB-101 in Patients With FTD

05:32

Supplemental Data on the Efficacy of Fenfluramine to Treat Dravet Syndrome-Associated Seizures

09:13

Clinical Trials Reinforce Positive Data for Hemophilia Treatments

06:20

Pathophysiology of AL Amyloidosis and the Mechanism of Action of Investigational Drug

15:27

Living With Stiff Person Syndrome

13:56

Erdheim-Chester Disease: Diagnosis and Treatment Options

09:32

Results from a Phase 2 Trial of Pegcetacoplan in Patients With C3G and IC-MPGN

05:48

Analysis of Growth Hormone Replacement Therapy in Patients Under Age 60 Years

08:44

Oral Octreotide in Patients With Acromegaly

09:29

Clinical Trials Testing Osilodrostat for the Treatment of Cushing’s Disease

06:30

Overview of Langerhans Cell Histiocytosis (LCH)

08:00

Recordati Rare Diseases

06:43

Romosozumab for Osteogenesis Imperfecta (OI)

04:30

Post Hoc Analysis of Levoketoconazole in Patients With Cushing’s Syndrome

04:21

Overview of Hypoglycemia

06:40

Investigational Therapy for the Potential Treatment of Congenital Hyperinsulinism

13:20

Delay of Diagnosis for Prader-Willi Syndrome

10:34

Current Treatment Options for Patients With Cushing’s Syndrome

09:31

Clinical Trial of Novel Octreotide Formulation for the Treatment of Acromegaly

05:16

Overview of Erdheim-Chester Disease (ECD)

07:09

Psychosocial Support for Patients With Lipodystrophy

12:14

Patient Perspective: Challenges of Lipodystrophy

08:29

Positive Results in Trials of First Oral Investigational Drug for Acromegaly

24:35

Signs and Symptoms of Fabry Disease, Featuring Nicola Longo, MD (Podcast)

15:14

Fabry Disease From a Patient’s Perspective, Featuring Maya Kineen (Podcast)

24:28

Fabry Disease Overview, Featuring William Burns, MD (Podcast)

07:57

Diagnosing Fabry Disease