Linda Montgomery • August 8, 2024
How patients and families can help build an understanding of HCM care, awareness, and advocacy

Clinical Trials:

Innovation has come to HCM through new therapeutic options under investigation. Several trials are open, and several more are planned for later this year or early 2025. 

So, what is a Clinical Trial?

A Clinical trial is a research study that tests a medicine, surgical, or behavioral treatment to see if it works.  With the data from these trials, researchers determine if a new treatment is safe and effective in people.  Research and its results help set the foundation for new research. When a medication is proven safe and effective for a group of patients, it may be considered for study in another population. By volunteering for a trial, we learn more about HCM and how we can live better with it, and maybe someday eradicate it all together.

Many scientific advances come from passionate patients and people like you participating in trials like the ones we have coming up.  

How do I learn more about clinical trial opportunities?

  1. HCMA has made it easy through our online tool   https://bit.ly/46v4d5D – Antidote, a health company platform for recruiting HCM patients that searches HCM trials in a friendly format.  The source of their data is Clinicaltrials.gov. 
  2. clinicaltrials.gov –  is a website listing clinical research studies and information about their results.
  3. Call the office (973) 983-7429 or email Linda@4hcm.org to learn more.

Other types of engagements are available. 

Sponsor-initiated Market Research Engagement: 

The HCMA has several opportunities annually for unique engagements to share the patient and family member’s lived experience to help improve outreach and marketing efforts. Sometimes, these engagements are disease awareness-focused and other times, they may provide “branded” marketing feedback on an approved class of therapeutics. 

So, what is a Sponsor-Initiated Market Research Engagement?

The sponsor-initiated marketing research engagement aims to gather and deeply understand information regarding HCM, its patients, caregivers, and behaviors. They are often interview-style (question-and-answer) phone conversations.   These engagements tend to be smaller in number with a quick turnaround.  For example, we may have 20 positions and must complete the activity in 4 weeks.  Other times, the information may be collected in a survey or a focus group meeting.  Most engagements will compensate you for your time. These opportunities typically include payment for service. To ensure we know you are interested in this type of engagement visit; https://www.surveymonkey.com/r/5YPBF7B

  Advisory board recruitment

So what is an Advisory board and what are their responsibilities?

An advisory board has several key responsibilities contributing to an organization’s success. Their primary role is to provide strategic advice and guidance to the management team. They use their professional or personal expertise and/or industry knowledge to help the organization make informed decisions and develop effective strategies.  

HCMA Blog

Medical Management May

April 30, 2025
Medical Management means using medicines and other non-invasive therapies to relieve symptoms. It's exciting that new treatments for HCM are being studied and that the FDA approved Camzyos in 2022! HCM can cause various symptoms depending on how stiff the heart is, how much scarring (fibrosis) there is, and whether there is obstruction. Some people have no noticeable symptoms. Common symptoms are chest pain/pressure, shortness of breath, lightheadedness, fatigue, palpitations, and brain fog. Syncope (fainting) is dramatic but not as common as the other symptoms. Medications are the first-line treatment for symptoms of HCM. If patients can't take one class of medication, they can try a medication from a different class. Here are some medications used to treat HCM and what they're for: Beta Blockers - make your heart beat slower and relax better. They allow the heart to fill more completely between beats. They can lower blood pressure and relieve symptoms. Calcium Channel Blockers - used to lower blood pressure and slow the heart rate. May reduce symptoms from obstruction in HCM. Myosin Inhibitors - cause the heart to beat with less force. They may reduce obstruction and septal thickness in some patients. Camzyos (mavacamten) is the first of this class of medication. For more information, click HERE . Antiarrhythmics - help to restore normal rhythm in the heart. Blood Thinners - anticoagulants and anti-platelets make it harder for blood to clot. They are prescribed to reduce the risk of stroke. This risk is higher in patients with afib, aflutter, and artificial heart valves. Diuretics , or "water pills," help the kidneys get rid of extra water and salt, reducing swelling in the body. Sodium Channel Blockers - may reduce gradient in obstructive HCM and can treat chest pain. Some also act as antiarrhythmics. Antibiotics - treat or prevent bacterial infections. In HCM, they may be given before dental work to prevent infective endocarditis. To learn more and see the side effects of these medications, click HERE .
On a green background, a line drawing of a head that has complicated arrows pointing outwards

Decisions in HCM: Why are they so hard?

By Gordon Fox • April 25, 2025
Part 2 of a series on making decisions in HCM. A key reason is that decision making is so difficult is that most decisions involve more than one risk.
Hypertrophic Obsctructive Cardiomyopathy

Camzyos® receives manufacturing and marketing approval for HOCM in Japan

By Erica Friedman • April 22, 2025
Bristol Myers Squibb announces that Camzyos has been approved for Obstructive Hypertrophic Cardiomyopathy in Japan.
More Posts