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Treatment

There are many types of monogenic diabetes, each due to mutations in different genes. Some types of monogenic diabetes can be treated with oral medication, while others are treated with insulin. Rarely, monogenic diabetes does not require any treatment, though monitoring by a physician is still important.

The most common causes of neonatal diabetes may be amendable to specific pills rather than insulin.  Lilly Jaffe was diagnosed with a type of monogenic neonatal diabetes due to a mutation in the KCNJ11 gene.  She was able to transition from multiple daily injections of insulin to a pill.  Her doctors began by giving her doses of sulfonylurea—a drug developed decades ago to enhance insulin secretion for Type 2 patients—and gradually reducing her insulin. Nine days later, she was able to completely stop taking insulin.

Unfortunately, not all people with neonatal diabetes due to gene mutations that typically respond to sulfonylurea pills will be able to successfully switch from insulin to pills.  Any attempt to change diabetes treatment should only be done under the direction and close supervision of an experienced doctor.

MODY due to mutations in HNF1A or HNF4A also commonly respond well to sulfonylurea pills.  In fact, people with these types of MODY are usually quite sensitive to sulfonylureas and thus, should be started on low doses even if their diabetes control was suboptimal on insulin or other types of medications.  Sulfonylureas are considered the treatment of choice for HNF1A-MODY due to many studies proving their effectiveness.

MODY due to mutations in the GCK gene generally require no treatment at all, though there are exceptions.

Just as in neonatal diabetes, not all people with HNF1A-MODY or HNF4A-MODY will be successfully treated with sulfonylurea pills.  Again, any attempt to change diabetes treatment should only be done under the direction and close supervision of an experienced doctor.

The best therapy in several types of neonatal diabetes and MODY remains unclear and seems to vary person-to-person.

People with monogenic forms of diabetes still need to check their blood sugar levels.  However, they may not need to check as often as a person with Type 1 diabetes.

For more general information about diabetes and its treatment, visit NIH’s National Diabetes Information Clearinghouse.