01:12

NORD 2025 Breakthrough Summit

10:39

HDFN: Outcomes of Intrauterine Transfusion and Patient Experiences

14:26

Rilzabrutinib Approval for Adult Patients With Immune Thrombocytopenia

09:55

ISUOG World Congress 2025: Hemolytic Disease of the Fetus and Newborn

07:43

Open-Label Extension Data of Del-Zota for Patients With Duchenne Muscular Dystrophy

13:04

Zopapogene Imadenovec in Treating Patients With Recurrent Respiratory Papillomatosis

12:49

Neuroblastoma: Beat Childhood Cancer Research Consortium

18:52

Neuroblastoma: New Approaches to Neuroblastoma Consortium

50:57

Case Studies in Diagnosing and Managing FOP

03:13

Updates in Refractory Chronic Cough From ERS 2025

24:17

Neuroblastoma: The Children’s Oncology Group

06:00

Results From the PEGASUS Clinical Trial of Pegvaliase in Patients With PKU

05:16

Plans for Phase 2/3 Clinical Trial of Bexmarilimab Plus SOC in Patients With MDS

10:37

Results From the LINKER-SMM1 Trial in Patients With High-Risk Smoldering Multiple Myeloma

06:16

New Staging Tool for Cutaneous T-cell Lymphoma (PROBEinCTCL)

09:10

Patient Perspective: TUBB4B and The Need for Awareness

10:17

Biomarker Validation in Niemann-Pick Disease Type C

22:45

Sophie’s Hope Foundation: A GSD1b Patient Advocacy Organization

11:45

Treating NF1-PN With Mirdametinib

08:27

Treating Rare Lung Cancer (ROS-1 Positive NSCLC) With Taletrectinib

06:43

Long-Term Safety and Efficacy Data on Givinostat for Patients With Duchenne Muscular Dystrophy

27:11

Immune Thrombocytopenia (ITP) Research Highlights: ISTH 2025

25:17

Immune Thrombotic Thrombocytopenic Purpura (iTTP) Research Highlights: ISTH 2025

44:48

Hemophilia Research Highlights: ISTH 2025

05:42

Patient Perspective: Lipodystrophy Diagnostic Journey

12:52

CAHtalyst Clinical Trials in Adults With Congenital Adrenal Hyperplasia

05:59

Diagnosis and Management of Hypoparathyroidism

08:04

Daily Symptom Burden of Hypoparathyroidism

09:04

Case Report: Patient With Congenital Adrenal Hyperplasia

19:58

XLH Community Impact Survey: Effects on Mental Health

01:35

Naxitamab Combination Therapy for Patients With Neuroblastoma

40:47

Myasthenia Gravis Clinical Research Highlights: AAN 2025

13:43

Patient Perspective: Diagnostic Journey and Challenges of Lipodystrophy

08:36

Long-Term Safety and Efficacy Results of Palopegteriparatide in Patients With Hypoparathyroidism

08:23

Case Report: Alemtuzumab-Induced Thyroid Eye Disease

09:17

What Clinicians Need to Know About Fibrodysplasia Ossificans Progressiva

06:19

Radiopharmaceutical Treatment for Neuroendocrine Tumors

04:08

Trends and Social Determinants of Teprotumumab in Thyroid Eye Disease

06:15

SMAshing My Limits

10:54

Diagnosing and Treating a Patient With Castleman Disease

11:19

Unmet Needs of Patients With Cushing’s Syndrome

04:56

Education Campaign for Thyroid Eye Disease

09:11

Recordati’s Presentations at ENDO 2025

06:05

Atumelnant for the Treatment of Congenital Adrenal Hyperplasia

05:14

Mental Health in Patients With Acromegaly

05:19

Disparities Between Biochemical Control and Symptom Burden in Patients With Acromegaly

10:26

Recordati Rare Disease Initiatives

07:45

Data on Paltusotine for the Treatment of Acromegaly

14:14

XLH Community Impact Survey: Overview and Results

01:06

CheckRare: Rare Diseases Are Our Focus, Expertise, and Passion

07:37

Mastocytosis Control Test: Implications for Physicians

08:35

New Staging Tool Available for Healthcare Providers Treating Patients With CTCL

15:43

Impact of Approval of Pegcetacoplan for Rare Kidney Diseases

09:21

Clinical Experience With Iptacopan for Treatment of PNH

06:50

Linvoseltamab in the Treatment of Relapsed/Refractory Multiple Myeloma

06:56

Evaluating Fenfluramine in Patients With Lennox-Gastaut Syndrome

20:30

Unmet Medical Needs in Rare Diseases

05:39

Results From Amivanatamab Clinical Trial for Metastatic Colorectal Cancer

08:51

Outpatient Administration of Naxitamab in Patients With Neuroblastoma

07:23

The ARISE Cancer Consortium