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Rare disease stocks
We cover 54 companies grouped under the Rare disease theme. The median market value in the group is $6.10B. Across the group, the median move over the past trading day is −0.13%. 79% of the 52 with a 200-day average trade above it. Price change over the past year runs from −13.0% to +206.9% across the middle 80 percent of the group, with a median of +63.9%.
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Table
54 of 268 companies match these filters.Clear filters
| Ticker | Company | Price | 1M return | YTD return | 1Y return |
|---|---|---|---|---|---|
| VRTX34.SA | Vertex Pharmaceuticals Incorporated | 88.56 BRL | +14.82% | −85.60% | −83.18% |
| VRTX | Vertex Pharmaceuticals Incorporated | $547.29 | +11.60% | +21.05% | +40.66% |
| ARGX | argenx SE | $1,047.10 | +20.05% | +24.66% | +47.90% |
| A1RG34.SA | argenx SE | 209.16 BRL | +14.71% | +11.37% | +36.97% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $239.00 | −17.13% | −40.28% | −47.38% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | 62.25 BRL | −15.31% | −44.14% | −49.35% |
| INSM | Insmed Incorporated | $124.39 | +19.46% | −29.77% | −7.54% |
| ROIV | Roivant Sciences Ltd. | $36.75 | +8.60% | +67.58% | +206.51% |
| JAZZ | Jazz Pharmaceuticals plc | $249.29 | −2.97% | +43.97% | +100.35% |
| ASND | Ascendis Pharma A/S | $250.25 | −1.65% | +17.33% | +29.66% |
| BBIO | BridgeBio Pharma, Inc. | $79.37 | −7.22% | +1.45% | +60.05% |
| CORT | Corcept Therapeutics Incorporated | $119.80 | +27.19% | +213.61% | +71.81% |
| BMRN | BioMarin Pharmaceutical Inc. | $65.61 | +5.70% | +10.36% | +11.85% |
| B1MR34.SA | BioMarin Pharmaceutical Inc. | 166.77 BRL | +12.08% | −0.14% | +4.97% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $89.12 | +3.89% | +31.46% | +308.99% |
| CYTK | Cytokinetics, Incorporated | $79.20 | −0.31% | +28.30% | +109.36% |
| IONS | Ionis Pharmaceuticals, Inc. | $63.36 | +14.58% | −20.47% | +48.21% |
| KYMR | Kymera Therapeutics, Inc. | $125.55 | +18.26% | +72.55% | +197.09% |
| PTGX | Protagonist Therapeutics, Inc. | $151.50 | +10.71% | +73.78% | +146.62% |
| IMVT | Immunovant, Inc. | $43.89 | +12.83% | +69.39% | +189.13% |
| CRNX | Crinetics Pharmaceuticals, Inc. | $84.84 | +1.17% | +81.55% | +179.08% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $111.87 | +3.54% | +6.53% | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $60.04 | +21.61% | +40.28% | +74.18% |
| ORKA | Oruka Therapeutics, Inc. | $99.44 | +9.94% | +251.75% | +555.07% |
| LQDA | Liquidia Corporation | $71.36 | −17.81% | +127.26% | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $67.12 | +20.05% | +69.84% | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $110.76 | +9.01% | +179.13% | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $72.69 | −3.58% | −5.28% | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $78.68 | −0.96% | +87.31% | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $98.51 | −12.22% | +26.18% | +31.96% |
| GRFS | Grifols, S.A. | $7.90 | −1.25% | −14.50% | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $26.36 | +1.78% | +42.49% | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $44.38 | +9.99% | +86.86% | +207.13% |
| MANE | Veradermics, Incorporated | $102.80 | −7.84% | +172.32% | +172.32% |
| AMLX | Amylyx Pharmaceuticals, Inc. | $37.08 | +89.67% | +224.69% | +292.38% |
| CPRX | Catalyst Pharmaceuticals Inc | $31.49 | 0.00% | +36.03% | +55.97% |
| CLDX | Celldex Therapeutics, Inc. | $40.57 | +14.02% | +50.20% | +79.51% |
| IRON | Disc Medicine, Inc. | $82.67 | +10.74% | +4.53% | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $25.14 | −11.54% | −13.31% | +60.64% |
| VRDN | Viridian Therapeutics, Inc. | $24.26 | +24.35% | −20.93% | +30.71% |
| VERA | Vera Therapeutics, Inc. | $36.82 | +10.50% | −24.27% | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $26.44 | +1.77% | +12.03% | −12.10% |
| PHVS | Pharvaris N.V. | $37.00 | +9.97% | +39.73% | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $17.92 | −2.77% | +50.71% | +124.00% |
| NKTR | Nektar Therapeutics | $73.77 | +4.24% | +69.90% | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $9.83 | +0.92% | +31.07% | +15.38% |
| COAG | Hemab Therapeutics Holdings, Inc. | $49.19 | +4.08% | +44.68% | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $25.89 | −3.93% | −27.50% | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $154.33 | +8.04% | +53.90% | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $16.54 | +10.56% | +7.75% | +36.58% |
| ADMA | ADMA Biologics, Inc. | $9.63 | +9.56% | −46.14% | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $33.04 | +2.32% | −4.23% | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $33.09 | +12.59% | +7.09% | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $33.99 | −5.56% | +25.06% | −13.45% |
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.