The Rare Disease Channel

Explore the latest updates and expert interviews
Rare Leukemias Rare Lymphomas Rare MPNs Bleeding Disorders Thalassemia Anemia Enzymopathies Paroxysmal Nocturnal Hemoglobinuria Plasma Cell Dyscrasias

Welcome to The Rare Disease Channel

Explore the latest updates in rare diseases, including expert interviews from major international congresses, podcasts and e-learning.

Latest Videos

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2:29
Unmet clinical needs in BPDCN: improving diagnosis and treatment approaches
Livio Pagano • 16 Mar 2023
1:53
Synergistic combination of RAP-GRL & TMPRSS6-ASO in β-thalassemia
Stefano Rivella • 11 Dec 2022
8:50
Updates from the HOPE-B trial: etranacogene dezaparvovec in adults with hemophilia B
Steven Pipe • 12 Dec 2022
6:11
HAVEN 7: emicizumab prophylaxis for the treatment of infants with severe hemophilia A
Steven Pipe • 12 Dec 2022

Rare Leukemias

Blastic plasmacytoid dendritic cell neoplasm
Acute Promyelocytic Leukemia
Chronic Myelomonocytic Leukemia
T-cell prolymphocytic leukemia
2:29
Unmet clinical needs in BPDCN: improving diagnosis and treatment approaches
Livio Pagano • 16 Mar 2023
1:57
Updates on treatment approaches for patients with BPDCN
Giovanni Marconi • 25 Oct 2022
4:41
Novel treatment approaches in BPDCN: the promise of CD123-targeting allogeneic CAR-T cells
Marina Konopleva • 29 Sep 2022
1:07
An insight into the CADENZA study: pivekimab sunirine in patients with R/R BPDCN
Naveen Pemmaraju • 27 Aug 2022
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1:45
New research areas in the treatment of AML
Naveen Pemmaraju • 11 Apr 2022
3:12
Advances & unmet needs in APL
Firas El Chaer • 23 Apr 2022
1:49
The efficacy of ATO and ATRA for APL
Mark Levis • 30 Sep 2021
3:20
Differentiation syndrome with lower-intensity therapy for AML
Amir Fathi • 24 Jun 2021
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2:36
Phase II study of E7820 in patients with splicing factor-mutant myeloid malignancies
Jan Philipp Bewersdorf • 11 Dec 2022
1:32
Phase II efficacy and safety results of ruxolitinib for patients with symptomatic CMML
Eric Padron • 9 Dec 2022
0:56
CMML highlights from ASH 2022
Eric Padron • 9 Dec 2022
1:46
Epigenetic dysregulation in ASXL-1-mutant CMML
Moritz Binder • 26 Aug 2022
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3:55
Pathobiology & treatment of T-PLL
Marco Herling • 12 Jun 2022
2:58
Itacitinib plus alemtuzumab for patients with T-PLL
Tapan Kadia • 10 Jun 2022
1:55
PET scans in NHL
Sally F. Barrington • 9 Jun 2021
1:26
Ruxolitinib plus venetoclax for T-PLL: rationale and findings
Charles Herbaux • 25 May 2021
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Rare Lymphomas

Burkitt Lymphoma
Waldenström’s
Cutaneous B-cell lymphoma
Cutaneous T-cell lymphoma
NK/T-cell lymphoma
Peripheral T-cell lymphoma
2:18
Phase I study of Bgb-11417 alone or with zanubrutinib in patients with NHL, WM, and CLL/SLL
Constantine Tam • 12 Dec 2022
2:51
Pirtobrutinib shows efficacy in BTKi refractory Waldenström’s macroglobulinemia patients
Chan Yoon Cheah • 9 Dec 2022
2:39
Antibody response to COVID-19 vaccine in patients with multiple myeloma and WM
Evangelos Terpos • 10 Dec 2022
1:33
Zanubrutinib in acalabrutinib-intolerant patients with various B-cell malignancies
Mazyar Shadman • 9 Dec 2022
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1:22
Diagnostics and treatment outcomes from primary CBCL
Amra Osmancevic • 15 Oct 2021
0:43
Classifying CBCL subtypes
Amra Osmancevic • 15 Oct 2021
1:05
The diagnostic work-up of CBCL in Sweden
Amra Osmancevic • 15 Oct 2021
0:29
Defining CBCL classifications
Amra Osmancevic • 15 Oct 2021
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2:24
Clonal evolution in patients with CTCL
Neha Mehta-Shah • 9 Dec 2022
1:12
TELLOMAK: lacutamab alone or with chemotherapy in patients with mycosis fungoides or Sézary syndrome
Neha Mehta-Shah • 9 Dec 2022
3:55
The standard of care in T-cell lymphoma: challenges and future outlooks
Swaminathan Iyer • 30 Sep 2022
3:08
The role of the microenvironment in T-cell lymphoma and the promise of pembrolizumab in this disease
Swaminathan Iyer • 10 Jun 2022
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0:42
Approaching NK/T-cell lymphoma
Won-Seog Kim • 11 Jun 2022
1:15
Results of Phase II study of tislelizumab in R/R NK/T-cell neoplasms
Emmanuel Bachy • 11 Jun 2022
3:56
Advances in treating T-cell lymphoma
Franco Cavalli • 24 Jun 2021
8:54
Advances in extranodal natural killer/T-cell lymphoma
Qing-qing Cai • 24 Jun 2021
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1:16
Highlights in PTCL at ASH 2022
Neha Mehta-Shah • 9 Dec 2022
1:36
Novel therapeutic regimens being explored in PTCL
Neha Mehta-Shah • 30 Sep 2022
2:01
Potential therapeutic targets in subsets of PTCL
Francisco Vega • 26 Sep 2022
3:22
How novel molecular techniques have improved and are providing more insight into the biology of PTCL
Francisco Vega • 26 Sep 2022
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Bleeding Disorders

Von Willebrand Disease

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1:34
WM and the clinical implications of acquired von Willebrand syndrome
Karan Chohan • 28 Oct 2022
0:59
Managing pregnant women with vWD
Susan Halimeh • 11 Jul 2022

Immune Thrombocytopenia

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4:20
ITP update: diagnosis, treatment & COVID-19 vaccines
James Bussel • 11 Oct 2021
1:26
Avatrombopag, rilzabrutinib and COVID-19 updates in ITP
Barbara Skopec • 28 May 2021

Anemias

Sickle Cell Disease

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1:59
Prospective study evaluating complement activation in patients with sickle cell disease
Eleni Gavriilaki • 12 Dec 2022
0:54
Long-term effects of haploidentical transplantation on pulmonary function in patients with SCD
Courtney Fitzhugh • 10 Dec 2022

Aplastic Anemia

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1:51
An overview of the pathophysiology of PNH
Shreyans Gandhi • 31 Mar 2022
1:27
Emerging treatments for BMF syndromes
Naveen Pemmaraju • 13 Dec 2021

Enzymopathies

Cold Agglutinin Disease

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2:54
Management of cold agglutinin disease
Sigbjorn Berentsen • 11 Oct 2021
6:28
Practice-changing potential from Cardinal: sutimlimab in CAD
Alexander Röth • 10 Dec 2019

Gaucher Disease

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0:49
Developing a patient-reported outcome instrument for Gaucher disease
Esther Oliva • 14 Jun 2018
1:22
Diagnosing and treating Gaucher disease
Derralyn Hughes • 16 Apr 2018

Plasma Cell Dyscrasias

Smoldering Myeloma

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1:51
Evaluating whether SARS-CoV-2 vaccination has an impact on risk of progression from MGUS to myeloma
Sigurdur Kristinsson • 12 Dec 2022
1:36
Multivariate model to predict the need for bone marrow sampling in patients with MGUS
Sigurdur Kristinsson • 12 Dec 2022

Amyloidosis

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0:44
Outcomes of patients with AL amyloidosis after failure of daratumumab-based therapy
Efstathios Kastritis • 11 Dec 2022
1:37
The importance of the multidisciplinary team in treating patients with myeloma and amyloidosis
Naresh Bumma • 11 Dec 2022
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