Recommended pre-submission checks
Powered by 

Topics Covered on Therapeutic Advances in Rare Disease
Therapeutic Advances in Rare Disease Journal Specifications
Indexed in the following public directories
Scopus
DOAJ
SJR
| Overview | |
| Publisher | SAGE Publishing |
| Language | English |
| Publication Time | 15 |
| Editorial Review Process | Blind peer review |
| General Details | |
| Language | English |
| Website URL | Visit website |
| Publication Details | |
| Editorial Review Detail | |
| Information for authors | |
| Author instructions | Visit website |
| Copyright Details | Visit website |
| Deposit Policy | Sherpa/Romeo |
| License type | CC BY, CC BY-NC |
| OA statement | Visit website |
View less
Planning to publish in Therapeutic Advances in Rare Disease ?
Upload your Manuscript to get
- Degree of match
- Common matching concepts
- Additional journal recommendations

Recently Published Papers in Therapeutic Advances in Rare Disease
Current therapeutic landscape of β-thalassemia: focus on gene therapy
- 28 Mar 2026
- Therapeutic Advances in Rare Disease
Reliability assessment using the test–retest method and minimal important changes in the Adult Fabry Disease Quality of Life Scale
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Challenges and opportunities for the use of telehealth in rare disease diagnosis, treatment, research, and education: key opinion leader interviews by the IRDiRC telehealth task force
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Patient and caregiver perspectives on neurodevelopmental and mental health care for RASopathies.
- 1 Mar 2026
- Therapeutic advances in rare disease
Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease.
- 1 Feb 2026
- Therapeutic advances in rare disease
Current therapeutic landscape of β-thalassemia: focus on gene therapy
- 28 Mar 2026
- Therapeutic Advances in Rare Disease
Reliability assessment using the test–retest method and minimal important changes in the Adult Fabry Disease Quality of Life Scale
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Challenges and opportunities for the use of telehealth in rare disease diagnosis, treatment, research, and education: key opinion leader interviews by the IRDiRC telehealth task force
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy
- 1 Mar 2026
- Therapeutic Advances in Rare Disease
Patient and caregiver perspectives on neurodevelopmental and mental health care for RASopathies.
- 1 Mar 2026
- Therapeutic advances in rare disease
Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease.
- 1 Feb 2026
- Therapeutic advances in rare disease