A dedicated cross-functional team of experts made up of strategic leadership and tailored support for rare clinical trials
We turn patient voices into better data.
We are focused on urgently accelerating the clinical development of new treatments in rare diseases.
Combining our industry knowledge, alongside personal experience within rare disease clinical trials, makes us a more valuable research partner for Sponsors looking for a truly tailored service experience. We can build a study around the individual patients’ needs, due to a nimble team that are never too far removed from the patient.
By providing a tailored approach to trial design and delivery, we can expedite the clinical development process and ultimately improve outcomes for patients and their families.
To speak with one of our rare disease experts about your trial or plans, please contact us.
We welcome your inquiries and will respond promptly.
As well as having a track record of supporting global research in cell and gene therapies and rare diseases in many therapeutic areas, we’re also experts in biostatistics, bioinformatics and adaptive/alternative study designs.
Acquired in 2022, Casimir (now Emmes Endpoint Solutions) takes a new approach to clinical studies from the patient’s perspective, enabling us to build a better understanding of rare disease progression and treatment.
Whether you are early clinical or post-marketing, we give you access to full-service protocol design, strategy and trial execution services.
Together, we design and run rigorous decentralized and hybrid studies designed to address the needs of regulators, payers and patients. We focus on getting to the truth of a disorder and adapting or developing the tools necessary to truly understand disease progression and treatment benefits.
A Sample of Emmes’ Experience in Rare Diseases
|Ophthalmologic Rare diseases including
inherited retinal diseases
|ABCA4 Retinopathy, Stargardt’s disease, Usher syndrome, Xlinked Juvenile Retinoschisis, Macular Telangiectasia Type 2, Retinitis Pigmentosa, Leber Congenital Amaurosis (LCA), Albinism, Best’s disease, Uveal Coloboma, Spinocerebellar Ataxia Type 7 (SCA7), Von Hippel Lindau, Behcet’s syndrome
|Blood Disorders including some blood cancers
|Sickle Cell disease, Aplastic Anemia, Acute Myeloid Leukemia, Multiple Myeloma, Myelodysplastic syndromes, Bone Marrow Failure syndromes
|Yellow Fever, Smallpox, Anthrax, Tularemia, Severe Acute Respiratory syndrome (SARS), Cytomegalovirus Infection, Neonatal Herpes (HSV)
|Immune System/Immune Deficiency
|Hemophagocytic Lymphohistiocytosis (HLH), Primary Immunodeficiency (PID), acute Graft-versus-Host Disease (aGVHD), Chronic GVHD, Steroid Refractory aGVHD
|AIDS-related lymphoma (e.g., Hodgkin and non-Hodgkin, Plasmablastic lymphoma (PBL), Burkitt’s lymphoma, Primary effusion lymphoma), Kaposi sarcoma, Anal cancer, Diffuse large B-cell lymphoma (DLBCL), T-Cell Lymphoma, other various rare leukemias/lymphomas
|Ulcerative Colitis, Primary Sclerosing Cholangitis
|Status epilepticus, Spasticity associated w/Cerebral Palsy
|Lysosomal Storage Disorders
|Fabry disease, Mucopolysaccharidosis II (MPS II or Hunter Syndrome), Niemann-Pick Type C, Cystinosis
|Other Rare diseases/disorders
|Bronchopulmonary Dysplasia (BPD), Limb-Girdle Muscular Dystrophies, Apnea of Prematurity