A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company’s new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale.
The ability to quantitatively monitor a patient’s health is an essential part of effective healthcare. A patient with “high blood pressure” couldn’t be adequately treated without knowing their systolic and diastolic blood pressure readings. An important marker for the progression of AIDS in an infected individual is their immune T-cell count – with this number, doctors and scientists can track the progression of the patient’s condition over time, as it either improves or worsens.
In short, before scientists can work on curing a disease, they need to develop a schema to monitor progression or regression of the condition. That’s where innovators like AMO Pharma come into play.
About Congenital Myotonic Dystrophy Type 1
Myotonic dystrophies are a kind of muscular dystrophy characterized by long-term muscular deterioration and weakness. Patients of myotonic dystrophies may occasionally experience prolonged, uncontrollable muscle contractions. Some muscles may not relax after use – someone gripping a pencil tightly in their hand may find themselves unable to release their grip on it.
Type 1 myotonic dystrophy is one of two forms of the genetically-linked condition. They lead to similar symptoms, though type 1 myotonic dystrophy is typically more aggressive and its symptoms are often more intense. They originate from mutations in different areas of the genetic code, and usually affect different regions of the body. Type 1 often affects the face, neck, hands, and legs below the knee; type 2 affects the neck, shoulders, elbows, and hips.
Congenital type 1 myotonic dystrophy is a severe form of type 1 myotonic dystrophy present from birth. Children with this form of the condition often have weak musculature, breathing problems, and intellectual disability.
About the Scale
The scale is elegant in its simplicity.
It’s derived from a simple Likert-style questionnaire administered to CMD1 patients. The questionnaire asks patients to self-evaluate their symptoms in a “strongly agree/disagree” style. The test would be fast and easy to administer, and takes into account the importance of the patient’s personal comfort levels.
The scale has shown promise as a low-burden assessor of CMD1 progression. AMO Pharma intends to use their new scale to evaluate the trial groups of its upcoming phase 2/3 study of children and teens with the condition. The rating scale will act as the primary outcome measure of the study – meaning it’s the standard against which the success or failure of the trial will be determined.
How does our ability to detect and observe a disease play a role in fighting it? Is developing rating scales more or less important than developing drug treatments? Why? Share your thoughts with Patient Worthy!