Phuturdex › Diseases › Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Drugs in development · updated 30 October 2026
Congenital, Hereditary, and Neonatal Diseases and Abnormalities: drugs in development
As of 30 October 2026 there are 5 active clinical trials (recruiting or ongoing, phases I–III) on 4 drugs for congenital, Hereditary, and Neonatal Diseases and Abnormalities. 4 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| TSHA-102 | III | 2 | NCT07480564 |
| Zeleciment Rostudirsen | III | 1 | NCT07608432 |
| Zodasiran | III | 1 | NCT07473843 |
| Senicapoc | II | 1 | NCT07282210 |
Sorted by most advanced phase and number of active trials.
All 4 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Specific forms
- Atopic dermatitis 91 drugs
- Sickle-cell disease 73 drugs
- Duchenne muscular dystrophy 35 drugs
- Hemophilia A 26 drugs
- Genetic disease 33 drugs
- Graves' ophthalmopathy 19 drugs
- Neurofibromatosis 1 28 drugs
- Cystic fibrosis 30 drugs
- Retinitis pigmentosa 25 drugs
- Beta thalassemia 34 drugs
- Eczema 27 drugs
- Wilms Tumor 37 drugs
Broader categories
- Nervous system heredodegenerative disease 3 drugs
- Genetic disease 33 drugs
- Mental disorder 24 drugs
- Neurobehavioral manifestation 8 drugs
- Nervous System Diseases 39 drugs
- Musculoskeletal disorder 22 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: NLM MeSH; NLM MeSH term: D009358 “Congenital, Hereditary, and Neonatal Diseases and Abnormalities”.