Rare Genetic & MetabolicUncontestedData as of 2026-09-04
Limb-Girdle Muscular Dystrophy is uncontested. Sarepta Therapeutics (SRPT) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with SRP-9003 in phase 3.
There is no public comparable to price it against. SRPT spreads its $2.4B market cap across 3 indications, so $809M of it is allocated here.
The one programme here is in late-stage development, but it carries no dated catalyst in the next 12 months.
$809M of allocated capital sits in Limb-Girdle Muscular Dystrophy, ranking it 161st of 299 indications by the money committed to it.
| Ticker | Company | Lead asset | Phase | Trials | Market cap | Pure play | Indications | Next catalyst |
|---|---|---|---|---|---|---|---|---|
| SRPT | Sarepta Therapeutics | SRP-9003 | Phase 3 | 3 | $2.4B | No | 3 | No dated event |
Sorted by phase, then by market capitalisation. Ticker links open the company profile.
Uncontested. One US-listed developer in the size band is running a clinical programme here, so there is no public comparable to price it against.
Rare Genetic & Metabolic holds $103B of the map's capital, 15.3% of the total, spread across 32 indications and 48 companies. 10 of those indications have more than one company competing.
See every Rare Genetic & Metabolic indication on the mapSarepta Therapeutics (SRPT, SRP-9003, Phase 3). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Limb-Girdle Muscular Dystrophy.
Of the 1 clinical-stage programme in Limb-Girdle Muscular Dystrophy, 1 is in Phase 3 or filed, 0 are in mid-stage development and 0 are in early-stage development.
No. SRPT is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Limb-Girdle Muscular Dystrophy, which is why the indication is classified as uncontested.
Allocated capital divides each company’s market capitalisation evenly across the indications it is running clinical programmes in, so every dollar of the universe is counted exactly once. Total market capitalisation competing counts a company in each of its indications and therefore sums to more than the universe.
Third-party market size estimates are not used anywhere on this page. Approved incumbents are excluded from competitor counts, and companies above $20B are outside the universe by construction.
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