Care for Rare:
Support the Rare Disease Act
A Signature Campaign for Orphan Disorder Support
What are Orphan, Rare Disorders?
A rare disease, otherwise called an orphan disorder is any health condition resulting from genetic defects that afflicts no more than 1 of every 20,000 individuals in the country. Rare disorders afflict babies in all socioeconomic levels. Besides the little information available about their nature, what makes dealing with rare disorders even harder is that they are long-standing, progressive, disabling and life threatening.
Filipino patients born with rare disorders (diseases) are “orphaned” by society. They suffer from social abandonment because of lack of existing network of support to aid them. Medical help is elusive under the conditions of the country’s health priority. The nature of their illness is hardly known due to lack of information and only a few medical professionals, particularly in the country, are aware of these disorders and know how to diagnose and address these conditions.
Pompe Disease, Gaucher Disease, Methymalonic Acidemia (MMA), Maple Syrup Urine Disease(MSUD), Fabry Disease, Galactosemia, , and Mucopolysaccharidosis are some of the sixteen RARE or ORPHAN metabolic disorders, which afflict Filipino children. There are about 165 documented cases of these RARE or ORPHAN disorders in the Philippines.
THE STORY OF “DICKOY”
Cynthia Magdaraog, the PSOD’s current president explains that her commitment to the advocacy was rooted in the fact that she herself is a parent of a patient with rare disorder. Her son Juan Benedicto, whom her family called “Dickoy’ is suffering from Pompe disease, a rare degenerative disorder characterized by the progressive deterioration of the muscles. “It could lead to death because the heart is also a muscle,” Magdaraog told The Manila Times.
Pompe disease patients do not expect to live very long but then the Magdaraogs saw a glimmer of hope. The United States Food and Drug Administration approved a form of enzyme therapy that could halt or minimize the progression of the disease. The expenses for orphan disorders are extremely prohibitive. “The cost of my son’s treatment, if we are going to pay for it, is P18 million a year,” Magdaraog reveals. She said that they were fortunate because some biological research companies were compassionate enough to give them free access to it.
Juan Benedicto today can only move his hands and is hooked to a respiratory ventilator 24 hours. But despite his present physical condition, the man is ripe with achievements. “Though battling with Pompe disease, my son is a productive individual,” Magdaraog narrates, adding, “He got an industrial psychology degree from De La Salle University and used to earn good money from servicing clients abroad.”
He is a successful entrepreneur who sells Apple computer products online and designs websites for clients local and foreign. The PSOD website, www.psod.org.ph, is a fine example of his work. In person, the man is disarmingly humble and personable. His works, both as a businessman, an advocate and a survivor speak well on his behalf.
The proud mother attested that her son is a patient-advocate. “He was invited several times to speak in behalf of the victims of rare disorders,” she narrates. Magdaraog reveals that one time, Juan Benedicto even helped a baby patient of Pompe disease to get access to treatment. “He even made sure that the baby be given treatment ahead of him,” Magdaraog says.
From “Leading the fight against rare diseases” by Perry Gil S. MallariManila Times, Nov.29, 2009
SUPPORT THE RARE DISEASE ACT OF THE PHILIPPINESThe RARE DISEASES ACT OF THE PHILIPPINES is a proposed legislation which provides the creation of the Rare Disease Program at the Department of Health. It will ensure:
Why Rare Disease Day?
Because we constantly need to raise awareness on rare diseases among decisions makers, health professionals and the general public. Information is key to improving living conditions for rare disease patients; raising awareness is therefore one of our primary goals
Because acting simultaneously in many places and in many countries can ensure the voice of rare disease patients is heard by more people
Because a day focussed on rare diseases can bring hope and information to people living with rare diseases, their carers and their families
Because we want equity in access to care and treatment for rare disease patients
Because we need an action that can bring all stakeholders of the rare disease community together with the same goal
Because we need more funds for research and care, and more research and efforts directed towards rare diseases
Because we need to keep fighting for rare disease patients
Because we need to coordinate policy actions at national level and at the
international level.
Rare Disease Day Themes
The international theme for the annual rare disease day celebration is prepared by the international event coordinator - the European Organisation for Rare Diseases (EURORDIS).
2012 Solidarity
Rare but strong together
2011 Rare Disease and Health Inequalities
Rare but equal
2010 Bridging Patients and Research
Patients and Researchers: Partners for Life
2009 Patient-centered Care
Patient Care: A Public Affair
2008 RD as a Public Health Priority
A Rare Day for Special People
Reference:
Philippine Society for Orphan Disorders
Rarediseaseday.org (Rare Disease Day Website)
Philippine Society for Orphan Disorders, Inc.
Telefax: 632 725-6519; Email: [email protected]
Address: 24-D Goldland Plaza 8 Eisenhower St.
Greenhills, San Juan, Metro Manila Philippines
visit www.psod.org.ph
A rare disease, otherwise called an orphan disorder is any health condition resulting from genetic defects that afflicts no more than 1 of every 20,000 individuals in the country. Rare disorders afflict babies in all socioeconomic levels. Besides the little information available about their nature, what makes dealing with rare disorders even harder is that they are long-standing, progressive, disabling and life threatening.
Filipino patients born with rare disorders (diseases) are “orphaned” by society. They suffer from social abandonment because of lack of existing network of support to aid them. Medical help is elusive under the conditions of the country’s health priority. The nature of their illness is hardly known due to lack of information and only a few medical professionals, particularly in the country, are aware of these disorders and know how to diagnose and address these conditions.
Pompe Disease, Gaucher Disease, Methymalonic Acidemia (MMA), Maple Syrup Urine Disease(MSUD), Fabry Disease, Galactosemia, , and Mucopolysaccharidosis are some of the sixteen RARE or ORPHAN metabolic disorders, which afflict Filipino children. There are about 165 documented cases of these RARE or ORPHAN disorders in the Philippines.
THE STORY OF “DICKOY”
Cynthia Magdaraog, the PSOD’s current president explains that her commitment to the advocacy was rooted in the fact that she herself is a parent of a patient with rare disorder. Her son Juan Benedicto, whom her family called “Dickoy’ is suffering from Pompe disease, a rare degenerative disorder characterized by the progressive deterioration of the muscles. “It could lead to death because the heart is also a muscle,” Magdaraog told The Manila Times.
Pompe disease patients do not expect to live very long but then the Magdaraogs saw a glimmer of hope. The United States Food and Drug Administration approved a form of enzyme therapy that could halt or minimize the progression of the disease. The expenses for orphan disorders are extremely prohibitive. “The cost of my son’s treatment, if we are going to pay for it, is P18 million a year,” Magdaraog reveals. She said that they were fortunate because some biological research companies were compassionate enough to give them free access to it.
Juan Benedicto today can only move his hands and is hooked to a respiratory ventilator 24 hours. But despite his present physical condition, the man is ripe with achievements. “Though battling with Pompe disease, my son is a productive individual,” Magdaraog narrates, adding, “He got an industrial psychology degree from De La Salle University and used to earn good money from servicing clients abroad.”
He is a successful entrepreneur who sells Apple computer products online and designs websites for clients local and foreign. The PSOD website, www.psod.org.ph, is a fine example of his work. In person, the man is disarmingly humble and personable. His works, both as a businessman, an advocate and a survivor speak well on his behalf.
The proud mother attested that her son is a patient-advocate. “He was invited several times to speak in behalf of the victims of rare disorders,” she narrates. Magdaraog reveals that one time, Juan Benedicto even helped a baby patient of Pompe disease to get access to treatment. “He even made sure that the baby be given treatment ahead of him,” Magdaraog says.
From “Leading the fight against rare diseases” by Perry Gil S. MallariManila Times, Nov.29, 2009
SUPPORT THE RARE DISEASE ACT OF THE PHILIPPINESThe RARE DISEASES ACT OF THE PHILIPPINES is a proposed legislation which provides the creation of the Rare Disease Program at the Department of Health. It will ensure:
- Provision for early and sustainable care for patients suffering from rare disease;
- Coordination of current activities of the DOH to provide patients and their families with better access to medical care, health information, and health products to treat their conditions;
- Support for public education and information campaigns and health professional training;
- Establishment and supervision of a system of research and development initiatives; and
- Coordination of resource generation efforts among relevant government agencies and private sector.
Why Rare Disease Day?
- Strengthen the voice of patients as one
- Give hope and Information to patients
- Bring stockholders close together
- Coordinate policy and action in different countries
- Get equity and access to care and treatment
Because we constantly need to raise awareness on rare diseases among decisions makers, health professionals and the general public. Information is key to improving living conditions for rare disease patients; raising awareness is therefore one of our primary goals
Because acting simultaneously in many places and in many countries can ensure the voice of rare disease patients is heard by more people
Because a day focussed on rare diseases can bring hope and information to people living with rare diseases, their carers and their families
Because we want equity in access to care and treatment for rare disease patients
Because we need an action that can bring all stakeholders of the rare disease community together with the same goal
Because we need more funds for research and care, and more research and efforts directed towards rare diseases
Because we need to keep fighting for rare disease patients
Because we need to coordinate policy actions at national level and at the
international level.
Rare Disease Day Themes
The international theme for the annual rare disease day celebration is prepared by the international event coordinator - the European Organisation for Rare Diseases (EURORDIS).
2012 Solidarity
Rare but strong together
2011 Rare Disease and Health Inequalities
Rare but equal
2010 Bridging Patients and Research
Patients and Researchers: Partners for Life
2009 Patient-centered Care
Patient Care: A Public Affair
2008 RD as a Public Health Priority
A Rare Day for Special People
Reference:
Philippine Society for Orphan Disorders
Rarediseaseday.org (Rare Disease Day Website)
Philippine Society for Orphan Disorders, Inc.
Telefax: 632 725-6519; Email: [email protected]
Address: 24-D Goldland Plaza 8 Eisenhower St.
Greenhills, San Juan, Metro Manila Philippines
visit www.psod.org.ph