Rare diseases are defined as conditions that affect less than one person in 2000. However, their large number, more than 8000 pathologies already identified, mean that the total number of patients often exceeds the number of cancer patients. More than 350 million people worldwide suffer from these pathologies.
One in 20 people are affected i.e. for Morocco 1.5 million patients affected by one of these pathologies. These diseases are often chronic, progressive and generally serious. Their expression is extremely diverse: neuromuscular, metabolic, infectious, immune, cancerous.
International Rare Diseases Day is organized every year on the last day of February in more than 90 countries. The main objective of this day is to raise awareness about rare diseases and their impact on the lives of those affected. On this occasion, theALLIANCE OF RARE DISEASES IN MOROCCO will organize his own “Rare Disease Day”, February 24, 2024 in Casablanca in the Marriot hotel. It will do this in partnership with the Sanofi laboratories and the Moroccan Association of Medical Biology (AMBM). Mr. Aziz DAOUDA will moderate the day’s debates, particularly during debates with the public and the press. IT SHOULD BE NOTED THAT MEDCURSUS AND OUJADACITY WILL PROVIDE THEIR SUPPORT TO THE EVENT BY BEING ONE OF THEIR MEDIA PARTNERS
A VERY WIDE AND SOMETIMES DISRUPTING PANORAMA OF RARE DISEASES
Extremely diverse – neuromuscular, metabolic, infectious, immune, cancerous… –80% of these diseases have a genetic origin. 3 out of 4 diseases start in childhood but some wait 30, 40 or 50 years before appearing..
They prevent you from: seeing (retinitis), breathing (cystic fibrosis), resisting infections (immune deficiencies), clotting the blood normally (hemophilia), growing and developing normal puberty (Turner syndrome: absence or abnormality in a girl of a of the 2 female sex chromosomes
Others cause: accelerated aging (progeria, 100 cases worldwide); repeated fractures (brittle bone disease) ; a transformation of muscles into bones (stone man disease, 2,500 cases worldwide); an uncertain gait, hesitant memory, behavior sometimes “inappropriate in relation to social conventions” and difficulty controlling the bladder (normal pressure hydrocephalus: accumulation of cerebrospinal fluid in the brain, after 60 years); anemia due to abnormal red blood cells (beta-thalassemia); cerebral sclerosis and progressive paralysis of all functions (leukodystrophy) … or even uncontrollable movements and intellectual weakening leading to dementia (Huntington’s disease).
The therapeutic journey is often chaotic for the sick. They often have difficulty obtaining the correct diagnosis, leading to sometimes dramatic delays in receiving treatment. And yet, if there is no curative treatment for most of these conditions, appropriate care can make it possible to “ living with a rare disease ».
PRIORITIES TO IMPLEMENT IN MOROCCO
People suffering from rare diseases pose innumerable problems in Morocco, particularly in terms of diagnosis and treatment. They also suffer from a lack of information, an absence of reference and competence centers as well as systematic neonatal screening for all newborns. They also suffer from a lack of universal medical coverage and the unavailability of certain medications, which further complicates their care..
The Alliance of Rare Diseases in Morocco (AMRM), has three MAIN CLAIMS aimed at:
– Rapidly grant long-term illness (ALD) status to a large number of rare diseases, particularly the most expensive ones first; this would thus guarantee systematic coverage and reimbursement of care at 100% of the social security reference base; It will be recalled that, at the moment, many patients are faced with reimbursement problems because they can only benefit from exceptional reimbursement on a case-by-case basis, having to be continually renewed and with the risk of suspension at any time;
– Establish an “orphan drug” status for therapies used in rare diseases, notably offering faster access to the market by shortening marketing authorization (AMM) deadlines; it could also be considered to exempt them from certain taxes and guarantee them commercial exclusivity over a certain period; in return for these advantages, public authorities should be able to negotiate price reductions on the most expensive medicines (some amount to several million dirhams!);
– Establish systematic neonatal screening aimed at detecting severe rare diseases of genetic origin, generally in babies: these include phenylketonuria, congenital hypothyroidism, congenital adrenal hyperplasia, cystic fibrosis and hemoglobinopathy.
Dr. MOUSSAYER KHADIJA الدكتورة خديجة موسيار Specialist in internal medicine and geriatrics in private practice in Casablanca. President of the Alliance Rare Diseases Morocco (AMRM) and the Moroccan association of autoimmune and systemic diseases (AMMAIS), Vice-President of the Moroccan Auto-Immunity Group (GEAIM).
TO KNOW MORE : INFORMATION STILL FRAGMENTAL FOR PATIENTS AS FOR DOCTORS, THE ALLIANCE OF RARE DISEASES IN MOROCCO (AMRM), AZIZ DAOUDA, AN EXCEPTIONAL TRAINER COMES TO THE SERVICE OF SICK ASSOCIATIONS
INFORMATION STILL FRAGMENTAL FOR PATIENTS AND DOCTORS
The therapeutic journey is often chaotic for patients even in developed countries like France! A survey carried out in 2015 by the Observatory of Rare Diseases highlighted numerous difficulties. One in five patients had a diagnostic delay equal to or greater than 6 years. Hospitalizations took place urgently for 45% of them. Examinations, care or treatments proved unsuitable for 57.5% of respondents. Finally, 90% of patients believe that health professionals have insufficient knowledge of this type of pathologies.
We also note that rare diseases considerably affect daily life. 51% of sick people and parents of children had to give up work. In 62% of cases, they trigger psychological or behavioral disorders and, for half, isolation from friends or family.
This observation observed in France is found in Morocco and much worse ! And yet, if there is no curative treatment for most of these conditions, appropriate care can make it possible to “live with a rare disease”.
THE ALLIANCE OF RARE DISEASES IN MOROCCO (AMRM)
Since its creation in 2017, the ALLIANCE has worked on a daily basis with many Moroccan associations specific to a given disease. It has also signed “strategic” partnership agreements with 12 patient associations: 1/ Rett Syndrome Association (AMSR), 2/ Association of Solidarity with the Children of the Moon in Morocco (ASELM), 3/ Association of spinal muscular atrophy (SMA), 4/ SOS Pku association, 5/ Moroccan association of hereditary angioedema patients (AMMAO), 6/ Moroccan Association for Child and Mother’s Health (AMSEM), 7/ Association SOS Marfantime (SOSM), 8/ Association Prader Willi Maroc (PWM), 9/ Flame of Hope Association for Autism and PKU Patients, 10/ Moroccan Association for Children Suffering from Osteoporosis (Osteogenesis Imperfecta) AMEOS , 11/ Moroccan Cystic Fibrosis Association (AMM), 12/ Fragile X Morocco Association (FxMa).
AZIZ DAOUDA AN EXCEPTIONAL TRAINER WORKS AT THE SERVICE OF SICK ASSOCIATIONSThe rare disease day organized on February 24 will be animated, notably during debates with the public by Aziz Daouda, technical and development director of the African Athletics Confederation. A graduate of the University of Bucharest and Montreal, this man is a specialist in sports training and the physiology of physical exercise, a subject he taught for years at the Moulay Rachid Institute. In this capacity he has directed numerous research projects, notably some in collaboration with the Ibn Sina University Hospital in the areas of diabetes and asthma. Aziz Daouda is best known for his career in sports. He held numerous responsibilities within the Ministry of Youth and Sports. His name remains above all linked to the field of athletics where he was, as a coach, manager and technical director, the mentor of the biggest stars of Moroccan athletics for around thirty years. Among other things, it allowed Morocco to be among the top 10 athletics nations for more than a quarter of a century.
Aziz Daouda is also involved in the development of sport for people suffering from rare diseases or disabilities. He thus intervened, in this capacity, during the first national Marfan syndrome day in December 2023 and at the celiac disease awareness day on February 10, 2024 where he chaired the event (event organized by the ASSOCIATION MOROCCAN FOR INTOLERANTS AND ALLERGICS TO GLUTEN (AMIAG).