Все публикации

0:14:40

African American with Huntington’s Disease Explains Her Diagnostic Journey

0:09:09

New Clinical Trial For Late Onset Pompe Disease Begins

0:13:58

Initiation of Biomarker Cohort in Clinical Trial for Facioscapulohumeral Muscular Dystrophy

0:10:47

Hereditary Angioedema Patient’s Preferences in Treatment

0:09:11

Initiation of the Astroscape Clinical Trial for TSC and FCD

0:10:06

Positive Interim Results From Study of IgA Nephropathy Treatment

0:04:01

Real World Treatment Data for Granulomatosis with Polyangiitis and Microscopic Polyangiitis

0:04:54

Results From Clinical Trial of Iptacopan in C3G

0:07:16

Real-Life Outcome Data With Risdiplam for Spinal Muscular Atrophy

0:07:24

The SYNAPSE-CMT Clinical Trial for Charcot-Marie-Tooth Disease

0:07:07

Results from the HIMALAYA Clinical Trial for Hepatocellular Carcinoma

0:08:43

Investigational Drug for Fragile X Syndrome Improves Cognition

0:06:29

FDA Approves Treatment for Chronic Graft Versus Host Disease

0:16:35

Advancements in the Treatment of Myasthenia Gravis

0:15:44

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

0:55:52

Patient Perspective: Journey to a Myasthenia Gravis Diagnosis

0:13:16

Breakthrough Therapy for CIDP Treatment

0:01:49

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

0:08:44

Oral Octreotide in Patients With Acromegaly

0:06:30

Overview of Langerhans Cell Histiocytosis (LCH)

0:08:00

Recordati Rare Diseases

0:04:18

Fabry Disease Patient Journey

0:15:29

How Rare Diseases Impact Daily Life: Scleroderma

0:04:22

Duchenne Muscular Dystrophy and Givinostat