Все публикации

0:07:56

Investigational Therapy For Duchenne Muscular Dystrophy

0:13:52

Newborn Screening For Lysosomal Disorders

1:00:16

LDRTC Symposium: Newborn Screening For Lysosomal Disorders

0:09:58

Chiesi Global Rare Diseases Report

0:07:20

IgA Nephropathy Treatment Options

0:05:26

Axonal Spheroids and Pigmented Glia (ALSP)

0:04:54

Diagnosing Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

0:01:32

Myasthenia Gravis Treatment Options

0:02:16

Zilbrysq for Treatment of Myasthenia Gravis

0:05:28

Antisense Therapy To Treat Huntington’s Disease and Spinocerebellar Ataxia

0:05:09

Recent Advances In Treating Advanced Kidney Cancer

0:11:31

The Limitations of Current Treatment Options for Huntington’s Disease and Spinocerebellar Ataxia

0:04:56

Lafora Disease Explained

0:09:11

Using Artificial Intelligence to Diagnose Rare Diseases

0:04:12

Multi-Omic Databases and Rare Cancers

0:01:33

Mitochondrial Myopathy

0:04:21

What is Sturge-Weber Syndrome?

0:01:55

Barth Syndrome Explained

0:03:17

VAMP2 Explained

0:03:29

This Patient with Sarcoidosis Was Misdiagnosed With Lung Cancer

0:02:37

KCNMA1-linked Channelopathy Explained

0:02:26

Sarcoidosis Explained

0:02:51

Tatton Brown Rahman Syndrome

0:04:05

Updates on Rozanolixizumab and Zilucoplan To Treat Myasthenia Gravis