Orphan Center of Morozov Hospital: 10 years of systematic assistance to children with rare diseases in Moscow

In 2014, on the instructions of the President of Russia, the first orphan center for children in the country was created in Moscow. The Morozov Children's Clinical Hospital became the base - the largest multidisciplinary children's hospital with 48 departments, 13 specialized centers, more than 1,100 beds (including 63 intensive care beds) and a highly qualified multidisciplinary team.

The creation of the center made it possible to build a sustainable model for routing, diagnostics and providing medical care to children with orphan diseases.   

• Victoria Sergeevna Lobanova, neurologist at the Center for Orphan and Other Rare Diseases at the Morozov Children's City Hospital, spoke about the development of the center at the II CIS Orphan Forum.

How the system developed:

1. Transfer of city neonatal screening to the hospital base
2. Creation of a medical genetics department
3. Formation of a multidisciplinary outpatient team (geneticists, pediatricians, endocrinologists, specialists in cystic fibrosis and other areas)
4. Development of a program for selective screening of hereditary metabolic diseases
5. Beginning of providing advisory assistance to children on referrals from outpatient clinics, including patients who are not screened (Later, mass screening of newborns for 11 diseases was introduced in Moscow, and then the list of both nosologies and methods was expanded)
6. The use of modern and unregistered drugs in Russia for severe metabolic conditions (hyperammonemia, homocystinuria, isovaleric acidemia, and others)

The center was the first in the country to perform infusion therapy for neuronal ceroid lipofuscinosis, and to begin treatment for SMA, cystic fibrosis, and Duchenne muscular dystrophy. A number of children began receiving therapy even before the advent of federal funding mechanisms.

Since 2021, after the creation of the Circle of Goodness foundation on the initiative of the President of the Russian Federation, the center has been providing support to Moscow children receiving treatment at the expense of the foundation.

In the first quarter of 2025, 1,038 children received assistance in Moscow at the expense of the fund, including:
— 248 with cystic fibrosis
— 146 with SMA
At the same time, therapy for patients with other orphan diseases not covered by the fund continues at the expense of the regional budget.
After the launch of the expanded neonatal screening program, Morozov Hospital became one of 10 interregional centers.

• Up to 17,000 newborns are examined monthly, and children from other regions (including Magadan Region and Chukotka)
• In 2024  122 patients with orphan diseases were identified as part of the screening

The functions of the center today:

• maintaining the regional segment of the federal registry (2,668 patients, including 1,388 children);
• dynamic monitoring of children with orphan diseases;
• submitting applications for drug provision, including applications to the Circle of Good Foundation;
• organizing and conducting medical commissions;
• telemedicine consultations with federal medical organizations;
• prescribing and conducting modern types of therapy (intravenous, intrathecal, replacement, gene, exon skipping);
• coordinating multidisciplinary care and distributing expertise across groups nosologies.

submitting applications for drug provision, including applications to the Circle of Good Foundation;
organization and holding of medical commissions;
telemedicine consultations with federal medical organizations;
prescribing and conducting modern types of therapy (intravenous, intrathecal, replacement, gene, exon skipping);
coordination of multidisciplinary care and distribution of expertise across groups of nosologies.

The Morozov Hospital Orphan Center is a model of a sustainable, integrated and clinically effective system of care for children with rare diseases. His experience shows how a scalable regional model can be built—from screening to high-tech care—in conjunction with federal policy.