communties for clinicians

Companies With Rare Diseases-Focused Platforms

This list includes major global companies as well as innovative biotech firms, all contributing significantly to rare disease research and treatment development.

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Pfizer Rare Disease**: [Pfizer Rare Disease](https://www.pfizer.com/science/rare-diseases)

Sanofi Genzyme**: [Sanofi Genzyme - Rare Diseases](https://www.sanofigenzyme.com/en/our-focus-areas/rare-diseases)

Novartis**: [Novartis Rare Diseases](https://www.novartis.com/our-focus/rare-diseases)

Shire (now part of Takeda): [Takeda Rare Diseases](https://www.takeda.com/what-we-do/rare-diseases/)

Roche**: [Roche Rare Diseases](https://www.roche.com/)

Amgen**: [Amgen Rare Diseases](https://www.amgen.com/)

Alexion Pharmaceuticals (AstraZeneca Rare Disease)**: Alexion - AstraZeneca Rare Disease](https://www.astrazeneca.com/our-therapy-areas/rare-disease.html)

Vertex Pharmaceuticals**: [Vertex Rare Diseases](https://www.vrtx.com/)

Gilead Sciences**: [Gilead Rare Diseases](https://www.gilead.com/)

Apellis Pharmaceuticals**: Focuses on ophthalmology, rare disease, and neurology. [Apellis Pharmaceuticals](https://apellis.com)

Enzyvant**: Specializes in rare diseases, known for FDA-approved drug RETHYMIC. [Enzyvant](https://enzyvant.com)Travere Therapeutics**: Focuses on treatments for rare kidney disorders, including IgA nephropathy.

[Travere Therapeutics](https://travere.com)

Pharnext**: Concentrates on developing therapies for neurodegenerative diseases, including Charcot-Marie-Tooth disease. [Pharnext](https://pharnext.com)Alchemab Therapeutics**: Develops antibody therapeutics for hard-to-treat diseases, including rare diseases.

[Alchemab Therapeutics](https://alchemab.com)iOnctura**: Develops therapies for difficult-to-treat tumors and rare diseases.

[iOnctura](https://ionctura.com)*PTC Therapeutics**: Specializes in therapies for rare diseases and has a comprehensive gene therapy center.

[PTC Therapeutics](https://www.ptcbio.com)Imara**: US biotech focusing on sickle cell disease and other hemoglobinopathies.

[Imara](https://imaratx.com)Keros Therapeutics: Develops therapies for rare hematologic and musculoskeletal disorders.

[Keros Therapeutics](https://www.kerostx.com)Dyne Therapeutics**: Concentrates on serious muscle diseases with their proprietary FORCE™ platform.

[Dyne Therapeutics](https://www.dyne-tx.com)

Dicerna Therapeutics**: Focuses on both rare and common diseases using RNA interference (RNAi) technology. [Dicerna Therapeutics](https://www.dicerna.com)AllStripes**: Research platform dedicated to empowering rare disease patients and communities in therapy development.

[AllStripes](https://www.allstripes.com)