Orphan Indications

Illingworth has a strong background in working in a variety of different orphan indications including Duchenne Muscular Dystrophy, Hunter and Hurler Syndrome and Graves Disease.

Orphan indications Illingworth has previously worked on include:

  • Von Willebrand Disease
  • Acquired Hemophilia A
  • Hunter’s Syndrome
  • AA Amyloidosis
  • Niemann-Pick Type C
  • Graves Disease
  • Fabry’s Disease
  • Pompe Disease
  • Acquired Thrombotic Thrombocytopenic Purpura
  • Nelson’s Syndrome
  • MELAS Syndrome
  • Cystic Fibrosis
  • Congenital Hemophilia A
  • Carcinoid Syndrome
  • Duchenne Muscular Dystrophy (DMD)
  • Myasthenia Gravis
  • Sanfilippo Syndrome
  • Isolated Hypogonadotropic Hypogonadism (IHH)

We are friendly yet proactive, logical yet empathetic and bring a human element to the enrolment and retention of patients (and their families) into clinical trials. We embrace patient centricity and always have the patient’s experience at the forefront of our minds when planning studies.

We have experience providing both CRO services and home research nursing, a highly attractive service in these complex, and often, distressing diseases. Offering the chance for patients to have their visits conducted within their own home alleviates the pressure on them and their family members. We work with both children and adult study subjects and maintain long term relationships with the family long after the studies complete.

The ability to offer home research nursing internationally enables more rare disease suffers to enroll in studies which otherwise would be too stressful or challenging for patients and their families. Illingworth strives to cater for individual needs in these rare disease trials and are happy to discuss the trial with the patient and family.

Please click on the link below for the case study.