Reference Public rare-disease knowledge base

Topic Hub

Therapy Development

Use this hub when the goal is to understand how FKRP therapeutic development is discussed without collapsing early signals into treatment claims.

1 guide · 1 page · 0 therapies · 1 paper · 4 studies · 0 glossary terms

Guides

Start with the site-level reference pages that define this topic.

Guide

For Researchers

This page is the research-oriented route into the FKRP site. It is designed to support literature capture, terminology alignment, and use of the paper and study libraries without assuming the user wants to start inside a database index.

GuideTherapy DevelopmentResearchersMixed

Papers

Use the paper summaries for dated evidence and limitations.

Paper Summary

Fukutin-Related Protein: From Pathology to Treatments

Review article mapping FKRP biology, disease mechanisms, model systems, and the treatment approaches that were emerging by 2021.

reviewTherapy DevelopmentGeneralMixed

Studies

Use the study records for current trial, registry, and cohort context.

Study Record

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

Phase 3 corticosteroid study record evaluating deflazacort in adults with FKRP-related LGMD2I, ultimately terminated early.

TrialTherapy DevelopmentFamiliesAdult

Study Record

ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.

TrialTherapy DevelopmentFamiliesAdult

Study Record

BBP-418 (Ribitol) FORTIFY Trial in LGMD2I/LGMDR9

Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9 and the most advanced current interventional program identified in this FKRP research sweep.

TrialTherapy DevelopmentFamiliesMixed

Study Record

Study of BBP-418 in Patients With LGMD2I

Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.

TrialTherapy DevelopmentFamiliesAdult

Pages

Use these site-level pages when you need practical support or field-wide status context.

Field Update

Therapeutic Pipeline

Current FKRP therapeutic pipeline with dated trial status, regulatory context, and explicit separation between trial records, sponsor updates, and historical programs.

Field UpdateTherapy DevelopmentFamiliesMixed