The MDA Journey – where do we go from here?
A Personal Vision

Background

I first learned about muscular dystrophy (MD) in early 2006 through a local radio program. I was immediately drawn to the muscular dystrophy cause. Since 2008, following the publication of my book “Voices of Hope – Coping with Muscular Dystrophy in Mauritius” I have had the privilege of being closely involved with the Muscular Dystrophy Association (MDA). With the support of dedicated Committee Members of the MDA, I embarked on establishing a new vision, mapping out a new path and laying the foundation stones for a new beginning at MDA. The ideas of the new vision stemmed from the latest medical and social management of muscular dystrophy in countries leading in that field. This account is a personal reflection on the recent journey of the Association.

The MDA has an affiliation of approximately 50 patients with muscular dystrophy; two-thirds of whom suffer from Duchenne from the age of 3-5 years, and about 10 adults with other types of muscular dystrophy. Information about the disease has been presented in articles on the www.mdamauritius.com website. The membership, including well-wishers who support with monthly membership contribution, amounts to about 100. In this account, persons with muscular dystrophy (MD) and their caregivers are described as “primary members.”

During my initial contact with the MDA in late 2006, and my first encounter with the patients directly afflicted by the impact of the incurable disease, as well as their caregivers, it was clear that the feelings and outlook of the patients and of their caregivers respectively about the disease were rather different. Many of the patients were not fully aware of the seriousness of the disease, as their caregivers did not share all information with them. The burden, pain and worries associated with the impact of the disease rested largely on the shoulders of the patient’s parents and caregivers. They often displayed a mixed feeling of powerlessness in the face of an incurable disease, worn-down by despair and resignation to their fate, and yet clung on to any glimmer of hope.

Many parents went through an ordeal of visits to numerous hospitals and doctors before being informed that their son has been diagnosed with Duchenne MD. The hardest blow was to be told: “I am sorry that nothing medically can be done for your son. Pamper him well.” Given that MD is a rare disease, and that many doctors in Mauritius, in both Government Hospitals and in the private sector have relatively limited knowledge and experience in its treatment, this was a harsh reality that the patients had to face. The statement that ‘nothing can be done’ has numbed the minds of many parents and for some, these words have even prevented them from understanding that there is still much that can be done to prolong their child’s life and enhance its quality. As such, some caregivers have been reserved in contributing to MDA’s New Vision.

Before mid-2008, people with MD in Mauritius were not treated for muscular dystrophy per se. Medical conditions, such as chest infections that many patients with MD endure, have been treated only on a specific case basis. To my knowledge, until the recent interventions by MDA, there has been no patient with MD who has received follow-up care or treatment from the moment the patient was diagnosed with the disease, to the fulltime confinement to a wheelchair, and eventually to the patient’s early death. Hospitalizing patients were rarely a source of relief as the medical personnel were untrained to cope with the situation. Caregivers were therefore left on their own to treat and care for their sons, only to dread the day when their son’s breathing and heart muscles would become affected beyond recovery.

The Ultimate Goal

Muscular Dystrophy foundations and associations in many parts of the world are working toward the same goal: a cure for muscular dystrophy. MDA, however, has had no contribution to offer on this front. Until a cure is found, interventions are put in place to slow down the progression of the disease. In line with the New Vision, MDA can strive to adopt similar medical and social interventions practiced in leading countries in MD management. MDA can strive to:

  • Be associated with the leading medical institutions, charities, and associations in muscular dystrophy world-wide, and draw benefits accordingly
  • Provide our patients with access to medical services that can delay the progression of the disease and enhance quality of life, while hoping to be greeted by a cure of the disease in the near future
  • Provide and facilitate social, educational and recreational services that enhance quality of life and sustain the overall well-being of people with MD
  • Provide appropriate information, skills, counseling and other support to the caregivers to manage the disease in the home, and to maintain physical and psychological well-being.

At the medical level, the ultimate goal is to mobilize the Ministry of Health (MOH) so that the Government Hospitals are equipped to manage patients with muscular dystrophy. This will require the training of medical personnel in understanding the disease and applying well-established protocols used in industrialized countries. However, realizing the potential challenges in mobilizing MOH, MDA’s New Vision has inevitably turned to the private sector to pioneer the New MDA mission.

The Road Travelled

In mid-2008, the MDA accomplished a major achievement with the provision of a motorized wheelchair to all of its needy patients. The motorized wheelchair transformed the patient’s quality of life overnight. It gave them a new sense of freedom and greater independence in their otherwise very restricted life.

In early 2009, for the first time in Mauritius, a team of 8 doctors joined MDA as volunteers to take on the challenge to begin treating Duchene patients – not to cure but to provide interventions that would slow down the progression of the disease. The doctors themselves humbly acknowledged that they were only making the first steps in understanding the disease and learning from what other countries were practicing by using existing protocols. This team soon expanded to include more doctors, three physiotherapists and one psychologist. My account entitled: “The doctors story” which appears on the website gives more details.

When Dr. Francois Cartault, an eminent geneticist from La Reunion Island, visited the MDA in November 2009, he was disturbed and shocked by the sight of the poor physical and health conditions of most of the Duchenne patients. He observed the twisted limbs, the deformed bodies, and the overall poor state of health of the patients at an early stage of the disease. When several patients passed away at the age of fifteen years in 2009, Idriss Cadjee, the past President of the Muscular Dystrophy Association in La Reunion had this to say: “In La Reunion, Duchenne patients do not pass away at the young age of fifteen.”

In Mauritius, orthopedic care to reduce the deformation of limbs and physiotherapy were not accessible to all patients. The latter were unaware of the benefits, and most could not afford such services on their own. There was no guidance on the benefits of proper nutrition for MD patients. Chest infection and the accumulation of phlegm that patients could not get rid of by themselves were often reported to be the cause of early deaths of Duchenne patients.

Ventilators

The first significant medical intervention of the New MDA was the lung function tests administered to approximately 15 Duchenne patients, in March 2008, at a private surgery. The examinations revealed the extent of the patient’s condition, and led MDA to procure its first ventilator – a variable positive airway pressured device (VPAP) that facilitates breathing. Scientific research has proven that this device enhances quality of life, and increases life expectancy by many years. The first VPAP costs us Rs 140,000, but subsequently, less sophisticated models at Rs 90,000 per machine were procured. Each Duchenne patient who has become permanently wheelchair-bound is likely to require such a machine. Three VPAPs were procured in 2008, followed by another 12 in 2009 and by 2010 approximately 25 patients have benefitted.

MDA had the good fortune of having Dr. M. Sundar, a member of our team of volunteer doctors, to manage the use of our first ventilators. The provision of vital and expensive ventilators to needy patients was only one side of the story. In an analysis by Dr. Sundar on the use of 14 VPAPs carried out in early 2010, it was troubling to find out that on average, compliance by patients on the use of the machine was a poor 45%, including under-utilization, and poor reporting by patients for medical review and maintenance of equipment. Several possible factors have been postulated for low compliance – (1) patients’ inadequate efforts to get adjusted to the use of the machine combined with the lack of caregivers support to facilitate the transition; (2) the feeling that since it was provided free, it had little benefits; (3) inadequate information and education transfer to the users and caregivers; (4) the haunting words of hopelessness that “nothing (medical) can be done for your son” still lingering in the minds of caregivers. This experience has been presented here in order to clearly demonstrate the challenge facing MDA in changing attitudes and behavior of primary members, despite offering well-tested technologies. The situation is currently being addressed with focus on better information, education and communication to the primary members.

In order to ensure high quality service, MDA has contracted in mid-2010 the monitoring and maintenance of all VPAPs to a specialized medical institution that retrieves all data on a monthly basis for MDA. The latter then analyses the data in respect of utilization and passes them on to the caregivers and the respective doctors for follow-up actions.

Medical Services

The volunteer team of medical professionals became a crucial foundation stone for the new MDA. Patients have been assigned to various doctors who provide free consultations at their respective surgery, administer vaccines, and follow up on the use of ventilators. The Treat-NMD protocol used in European countries to manage MD was made available to our team of doctors. Dr. F. Cartault’s visit provided a forum for the doctors to interact and share their experiences. This landmark visit paved the way for collaboration with the Association Française contre les Myopathies (AFM).

In mid-2010, Dr Cartault broke the amazing news of a trial treatment of Duchenne patients to be initiated soon by AFM. In that context, the blood samples of three Mauritian patients were dispatched for molecular biology tests. Having established a relationship with the AFM is naturally a remarkable accomplishment for the MDA, and a first step for MDA to be associated with global research.

Physiotherapy sessions have been initiated with groups of parents and patients, emphasizing their importance at an early stage of the disease in order to prevent early deformations of limbs and enhance the overall well-being of the patients. Training sessions on the removal of phlegm have also been imparted to caregivers. In early 2010, the officer-in-charge of the Orthopedic Centre at Coromandel, which is attached to MOH, gave a talk to the MDA primary members on the various types of orthoses offered free by the Centre. Only a few primary members had earlier benefitted from orthoses from the Centre. Examining the patients in the room, the speaker made the disturbing remark: “unfortunately, certain interventions are too late for many of the patients.”

Another important acquisition of the MDA was the two cough-assist machines, each costing Rs 350,000, which help to remove phlegm, the accumulation of which is a major contributory factor to the deaths of our patients. The challenge is for the caregivers to apply the knowledge diligently and to ensure the effective use of these machines by the patients.

Since 2009, Clinic Fortis-Darné has extended a helping hand by providing free cardio and lung function tests to all patients to celebrate World Health Day, and has since become a regular partner. Advice on nutrition was added in 2010. The Clinic is also subsidizing the cost of treatment for any in-patients.

With the support of the Ministry of Social Security, seasonal vaccinations have been administered to patients annually since 2009. MDA has provided pneumococcal vaccines which have been administered by MDA team of doctors in 2010.

My dialogue with Apollo Bramwell Hospital has added a new dimension to the services available to our primary members since early 2011. The Hospital will provide free of charge to all our patients an annual spine radiograph combined with orthopaedicians’ consultation. Based on the outcome, tendon and spinal surgery could be entertained for needy patients at preferential rates – this would be pioneering activities for the MDA.

Motorized Beds and Other Items

In mid-2010, fifteen medical beds with remote controls that can be operated by the patients were distributed to our primary members; seven more were received in January 2011. The response has been phenomenal by both patients and caregivers who have since seen a transformation in the patient’s quality of life. These beds have given the patients greater control of their lives, and have also largely reduced the burden experienced by caregivers in having to move their loved ones at all times. These items have also restored the dignity of patients. Other items, such as hygienic chairs have also enhanced the patients’ quality of life.

Educational Support

The Information, Education and Communication component of the medical interventions have been touched upon above. On the Education front, a large number of school-age patients have dropped out of school as well as specialized educational institutions. Sadly, some of them did not actually drop out of school by their own free will, but have been coerced or encouraged to leave school after only a few years of schooling. Efforts are being made to ensure that this trend is reversed through dialogue with the educational institutions.

Another of MDA’s priorities is to look for sponsors to provide laptops to more of our patients. Around the early age of 8 or 9 years, a Duchenne patient starts losing his ability to move his arms and hands, as his muscles progressively deteriorate. Writing or reading, which many of us take for granted, presents special challenges for any child who cannot properly lift or hold a book or even a pencil. Yet their mental ability is as sharp as ever, and their thirst for knowledge is as intense as the healthy boy next door. A few of our patients are at colleges and one is pursuing a university education. Laptops would allow our boys with MD to communicate, write, learn, read, and let their minds navigate well beyond the confines of their homes via the internet. Until now, MDA has provided about 10 laptops to its primary members.

Social Events

Organizing social activities has been a priority of the MDA as part of the holistic approach to managing the disease and promoting positive mental well-being for patients and caregivers. The events bring patients and caregivers together as a big family, and provide entertainment for all. Such events have included visits to the airport and getting on board an airplane, to the State House welcomed by the President and First Lady, Casella Park, annual end-of-year party, concerts and Charity Walks for fundraising. The State House visit in 2010 was an emotional outing for many primary members, young and adults alike, as they set foot on the Reduit grounds for the first time in their lives.

Building MDA’s Credibility and Partnership

Special efforts have been made to shape the MDA and demonstrate credibility in the eyes of primary members, as well as the community at large, particularly the donor community. Early on, certain ingrained practices had to be addressed to make way for improved procedures that embody effectiveness and transparency in the delivery of services. Empowered by its newly acquired knowledge on muscular dystrophy management, the New MDA was driven by the deep conviction and passion that the route chosen was the most appropriate; the translation of its visions into results and realities for our primary members were powerful selling points to sponsors. The credibility of MDA as an efficient organization with an admirable cause has therefore appealed to many sponsors, which includes many of the most respected business institutions in the country, as well as the Australian Government. At this junction, it is appropriate to acknowledge the generous contribution of our many sponsors and donors who have provided funds for MDA operations, including medical equipment, examinations and other related medical activities.

Programme and Delivery

While the Executive Board members define the vision and mission of MDA, a small Office was set up to translate the mission into realities for our primary members. It was crucial to recognize the respective role of each of the two entities – different functions and yet inter-dependent to form the whole. Problems were encountered on occasions when certain board members intruded into the domain of the Office, thus disturbing the strategic balance of the two bodies. Equally, staff members acting under the influence of board members, thereby compromising good business practices, were unfortunate learning experiences. As president since 2009, I was fortunate to be supported by dedicated members who were motivated by the prospects of giving muscular dystrophy patients a new beginning, despite some impediments both at the Board and Office levels, which, unfortunately, are far too common in some social organizations.

With the limitation of financial resources of MDA, the Office consisted of a part-time team leader assisted by a Project Assistant and two Field Counselors. The challenge was to apply as much of the theoretical knowledge gained as a matter of urgency, since a patient’s disease could only get worse as each month passes by. It was obvious that the learning curve for staff members would be very steep. Home counseling was introduced as an attempt to bring the New Vision to the patients and caregivers through information, education and communication. The experience has been rather mixed, as reflected in the changes or otherwise in attitudes and behavior of the primary members. However, the larger obstacle to the smooth formation of the Office was, unfortunately, the staff changes, particularly in the leadership role, due to various reasons.

Despite the hiccups experienced by the Office, staff members have gained confidence as they move along, and the programme expanded in both contents and dimensions. Looking objectively, the journey reached in a short span of time had been very commendable.

The Journey Ahead

The road ahead looks highly promising, propelled by the tangible results on several fronts and the keenness and confidence of the team to continue working for this cause. While the roadmap to reach certain set physical objectives would appear more straight-forward, the challenges are in the areas which require attitudinal and behavioral changes.

Optimal Medical Care

In countries where muscular dystrophy is managed proficiently, the disease is usually diagnosed at the Government Hospitals which are equipped with qualified personnel. In Mauritius, this would be the natural progression, and the urgency of the matter could not be ignored. The know-how gained by MDA so far through the magnanimous contribution of the medical personnel and institutions in the private sector should, hopefully, be a catalyst in mobilizing the political will of the Ministry of Health (MOH). My initial discussion with the MOH at the highest level in late 2009 looked encouraging. However, progress on that front has been slow. This avenue is still being pursued, as it is a critical link to ensure that muscular dystrophy moves into the mainstream of MOH concerns.

The MDA team of medical professionals would continue to gain more experiences while managing the conditions of the patients and learning from other countries. The team can venture beyond the confines of the patients and explore the genetic links in the family on the transmission of the disease, by screening potential carriers of the disease who are related to the MD families.

It is vital that MDA makes the best use of the existing services provided by its team of volunteer medical professionals, the medical institutions, namely Fortis Clinique Darné, Apollo Bramwell Hospital and the Orthopedic Centre at Coromandel, and the Ministry of Social Security on vaccinations.

Building the Capacity for Effective Programme and Delivery

The sustainability of the MDA depends largely on the effectiveness of the Executive Board as well as the Office. These two entities should co-exist in harmony, respecting each other’s role. In that respect, the primary and other MDA members should elect Board members who are committed and can inspire MDA. In the early days of the New MDA, as the Office builds its capability, the Board has largely been providing the vision and shaping the programme. However, as the Office accumulates experiences and enriches its programme and delivery systems, I foresee that this visionary role would be largely assumed by the Office; the Board would then be largely overseeing the performance of the Office and continue to play a key role in policy matters.

The Office should be run with great professionalism, eagerness to expand MDA’s horizon and monitor success in terms of improved quality of life for our patients and their caregivers. It should continue to focus on transparency and efficiency in the delivery of services, empowerment of primary members, building wider networking both locally and overseas, and creating a stronger membership base as well as effective dialogue with donors and sponsors for financial long-term sustainability. This would be achieved by a visionary leader supported by a team of high quality staff. With the fast expansion and the growing richness of the programme, the re-organization of the office staffing is urgently required and is being addressed.

The MDA Family

When I joined MDA in early 2008 as a full member, only six members turned up at the Annual General Meeting of 2008. Dissatisfaction expressed by primary members at the time included the lack of transparency in the management of resources and delivery of services and a lack of sense of purpose. My personal assessment was that, beside what seemed to be a top down approach of management, there were also inadequate opportunities for primary members to share their life experiences, to contribute, or to be heard.

In the recent years, I have happily noticed the much greater participation of the primary members, particularly at parents’ meetings and medical events. Unceasing efforts have been made to stimulate primary members to act as a supporting family member, irrespective of ethnic, religious and economic and social background. We are currently working on establishing an “Information & Knowledge Corner” in a section of the Office, that will provide MD families and visitors with audio-visual information and reading material on MD and the MDA cause.

At the end of the day, the flag bearers of MDA will always be those persons with muscular dystrophy and their caregivers. Hopefully, some well-wishers will cross their paths, join them in their march and help to take up their cause – providing the well-needed energy and selfless devotion until the goal is reached.

Philip Wan (February 2011)

Realizing our Mission:

We believe that the medical management and daily well-being of those it serves are of vital importance. In accomplishing our mission, we are committed to serve as:

  • The primary MD resource in Mauritius: Promoting the highest values, principles and practices to ensure accountability and effectiveness.
  • An effective advocate: Demonstrating our commitment to those living with MD, voicing the need for a positive quality of life today and in the future.
  • A respectful associate: Collaborating with the community, donors and sponsors; working within a network of organizations involved in the MD field; actively engaging volunteers, particularly family members with MD, to ensure that we maximize our collaborative efforts for the cause.