Parents Receive News That Their Child’s Niemann-Pick Disease Medication Will be Discontinued

Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk.

According to a recent article in NBC Los Angeles, Sara McGlockin, Marian’s mother, reports that Marian has received bi-monthly injections of the experimental drug Adrabetadex since 2017 and has made substantial improvements. Marian’s father called Adrabetadex their lifeline.

That is why the family was devastated when they received a letter of discontinuance several weeks ago from Mallinckrodt Pharmaceuticals. The drug manufacturer stated that they will no longer continue studying Adrabetadex because of the lack of potential benefits.

About Niemann-Pick

Niemann-Pick is classified according to its genetic cause, as well as being classified according to symptoms. The four types are A and B plus types C1 and C2. Types C1 and C2 are similar and are usually diagnosed in childhood.

Symptoms involve poor coordination and muscle tone, liver and lung disease. Patients often have speech difficulty and eventually experience problems swallowing.

A Band of Mothers

Another mother reacted to Mallinckrodt’s letter by saying that if they stop the drug it ends her daughter’s life.

Other parents of children with the disease agree with Sara McGlockin that their child’s progress provides proof of Adrabetadex’s benefit.

Tiffany Rubben’s son receives Adrabetadex injections. Tiffany reports that he is benefiting from the drug. He is able to ride a bike aided by training wheels and is able to participate in baseball games.

Mothers of children with Niemann-Pick throughout the country have come together in an all-out effort to keep Adrabetadex accessible. They are writing letters, conducting podcasts, and simply pleading for help.

Even the Children’s Hospital of Los Angeles (CHLA) that administers Marian’s shots is assisting the families of children with Neimann-Pick. Dr. Kim of CHLA agrees that Adrabetadex deserves further study as evidenced by the benefits Marian has received.

As a last resort, parents and doctors are hoping that another pharmaceutical company will fill the void.

NOTE: See message from Mallinckrodt Pharmaceuticals.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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