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Rare disease stocks
We cover 59 companies grouped under the Rare disease theme. The median market value in the group is $3.19B. Across the group, the median move over the past trading day is −0.61%. 70% of the 57 with a 200-day average trade above it. Price change over the past year runs from −74.9% to +222.7% across the middle 80 percent of the group, with a median of +60.6%.
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Table
59 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% | Nov 2, 2026 |
| ARGX | argenx SE | $65.51B | Large cap | +47.90% | Oct 22, 2026 |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% | Oct 29, 2026 |
| INSM | Insmed Incorporated | $27.16B | Large cap | −7.54% | Oct 29, 2026 |
| ROIV | Roivant Sciences Ltd. | $26.55B | Large cap | +206.51% | Nov 9, 2026 |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +100.35% | Nov 4, 2026 |
| BBIO | BridgeBio Pharma, Inc. | $15.52B | Large cap | +60.05% | Oct 28, 2026 |
| CORT | Corcept Therapeutics Incorporated | $12.95B | Large cap | +71.81% | Nov 4, 2026 |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% | Oct 26, 2026 |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +308.99% | Nov 24, 2026 |
| CYTK | Cytokinetics, Incorporated | $11.01B | Large cap | +109.36% | Nov 5, 2026 |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +48.21% | Nov 4, 2026 |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +197.09% | Nov 3, 2026 |
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% | Nov 5, 2026 |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% | Nov 9, 2026 |
| CRNX | Crinetics Pharmaceuticals, Inc. | $9.00B | Mid cap | +179.08% | Nov 5, 2026 |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% | Nov 3, 2026 |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% | Nov 12, 2026 |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% | Nov 11, 2026 |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% | Nov 3, 2026 |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% | Oct 29, 2026 |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% | Nov 5, 2026 |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% | Nov 5, 2026 |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% | Oct 27, 2026 |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% | Nov 4, 2026 |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% | Nov 5, 2026 |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% | Nov 5, 2026 |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% | Nov 12, 2026 |
| AMLX | Amylyx Pharmaceuticals, Inc. | $4.13B | Mid cap | +292.38% | Nov 5, 2026 |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% | Nov 9, 2026 |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% | Nov 5, 2026 |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% | Oct 27, 2026 |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% | Nov 5, 2026 |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% | Nov 5, 2026 |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% | Nov 3, 2026 |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% | Nov 12, 2026 |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% | Nov 12, 2026 |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% | Nov 12, 2026 |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% | Nov 2, 2026 |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% | Nov 16, 2026 |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% | Nov 10, 2026 |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% | Nov 11, 2026 |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% | Nov 9, 2026 |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% | Nov 4, 2026 |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% | Nov 12, 2026 |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% | Nov 3, 2026 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% | Oct 29, 2026 |
| GOSS | Gossamer Bio, Inc. | $85.01M | Micro cap | −92.93% | Nov 4, 2026 |
| SPRO | Spero Therapeutics, Inc. | $71.05M | Micro cap | −38.38% | Nov 12, 2026 |
| SCYX | SCYNEXIS, Inc. | $56.41M | Micro cap | +579.04% | Nov 4, 2026 |
| ATYR | aTyr Pharma, Inc. | $54.05M | Micro cap | −89.34% | Nov 5, 2026 |
| MCRB | Seres Therapeutics, Inc. | $47.81M | Micro cap | −74.79% | Nov 4, 2026 |
| MREO | Mereo BioPharma Group plc | $44.39M | Micro cap | −84.20% | Nov 9, 2026 |
| QNCX | Quince Therapeutics, Inc. | $31.84M | Micro cap | +100.71% | Nov 12, 2026 |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.24% | Nov 10, 2026 |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −94.18% | Nov 12, 2026 |
| INM | InMed Pharmaceuticals Inc. | $7.64M | Micro cap | −36.44% | Sep 23, 2026 |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% | Nov 12, 2026 |
| XRTX | XORTX Therapeutics Inc. | $4.03M | Micro cap | −43.99% | Nov 12, 2026 |
Covers ~22,000 companies across the US, Europe and Asia-Pacific (ex mainland China, Hong Kong and India), refreshed nightly. Every listed stock opens as a full analysis for $9.90.