Diamond-Blackfan anemia

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At a Glance

Live from database

Regulatory Watchboard

7 events
Mar 2026Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

Apriligen, Inc. — PHASE1

TrialNOT YET RECRUITING
Oct 2025Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)

Northwell Health — NA

TrialRECRUITING
Jul 2017Nonmyeloablative Stem Cell Transplant in Children with Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

University of Calgary — PHASE2

TrialRECRUITING
Jun 2016Investigation of the Genetics of Hematologic Diseases

St. Jude Children's Research Hospital

TrialRECRUITING
Mar 2013CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Diane George — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Sep 2004Diamond Blackfan Anemia Registry (DBAR)

Northwell Health

TrialRECRUITING
Nov 2001Cancer in Inherited Bone Marrow Failure Syndromes

National Cancer Institute (NCI) — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

Patient Assistance Programs1

Dyural 80-Lm

Advanced Rx Pharmacy of Tennessee, LLC

OpenContact for details

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Approved Treatments

1 FDA-approved

Dyural 80-Lm

(METHYLPREDNISOLONE ACETATE, LIDOCAINE HYDROCHLORIDE, BUPIVACAINE HYDROCHLORIDE, POVIDINE IODINE, ISOPROPYL ALCOHOL)Orphan drugstandard

Advanced Rx Pharmacy of Tennessee, LLC

12.1 Mechanism of Action Bupivacaine blocks the generation and the conduction of nerve impulses, presumably by increasing the threshold for electrical...

FDA label ↗

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Clinical Trial Landscape

6 active trials
1Phase 2
2N/A
2Unknown
1PHASE1, PHASE2
6Total recruiting
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Recent News & Research

No recent news articles indexed yet for Diamond-Blackfan anemia.
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Specialist Network

Top 6 by expertise

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