Patient Resources

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Learning from each other to help effect positive change in the rare-disease community

Catalyst is grateful to work with those who are affected with the rare diseases for which we are developing treatments. It is this spirit that has become our focus and drives our decisions and processes. We are committed to helping to communicate the points of view of those whose voices may not have been heard and whose needs have been overlooked.

As a small pharmaceutical company dedicated to the research and development of treatments for rare neurological and neuromuscular diseases, Catalyst is extending the reach and depth of our relationships with affected patients and their caregivers. As we continue to follow them on their challenging journeys, we’re dedicated to sharing what we learn from those who can help effect positive change—researchers, healthcare providers, insurance companies, policy makers, and advocacy organizations.

Catalyst pharmaceuticals—working toward a brighter future for everyone affected by a rare disease

Organizations dedicated to helping people with rare diseases

Catalyst Pharmaceuticals partners with advocacy organizations to support patients and the rare disease community. The following organizations can provide information and assistance in dealing with neuromuscular and other rare diseases.

National Organization for Rare Disorders (NORD)

Established in the late 1970s and early 1980s, NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them.  NORD, along with its more than 260 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. Learn more

Myasthenia Gravis Foundation of America (MGFA)

The Myasthenia Gravis Foundation of America is the only national volunteer health agency in the United States dedicated solely to the fight against myasthenia gravis. MGFA is committed to finding a cure for myasthenia gravis and closely related disorders, improving treatment options, and providing information and support to people with myasthenia gravis, through research, education, community programs, and advocacy. Learn more

Global Genes

Global Genes is a leading rare disease advocacy organization with global reach to the worldwide rare community of patients, caregivers, advocates, and clinical partners. Its mission is to eliminate the challenges of rare disease. Learn more

Living with LEMS

Part of the Global Genes community, LivingwithLEMS.org is a site for all patients with Lambert-Eaton myasthenic syndrome (LEMS) and their caregivers and family members that offers information, resources, and support. The goals of this site are to build connections and offer resources and support to help LEMS patients become informed advocates.  Learn more

Muscular Dystrophy Association (MDA)

Established in June 1950, the Muscular Dystrophy Association is committed to saving and improving the lives of kids and adults living with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related muscle-debilitating diseases that take away physical strength, independence, and life. Learn more

Child Neurology Foundation (CNF)

Established in 2001, the Child Neurology Foundation serves pediatric patients through advocacy, education, research, and support initiatives. Its mission is to serve as a collaborative center of education and support for caregivers and their children with neurologic conditions. Learn more

Disease Awareness and Events Calendar 

Throughout the year, organizations support a variety of initiatives to raise awareness and stimulate conversations about rare diseases. These events bring communities together to help educate the public and influence decision makers in an effort to affect real change in lives of those effect by rare diseases.

February

Rare Disease Day 

February 28

Established in Europe in 2008, Rare Disease Day is now observed in more than 65 countries including the United States Its focus is to highlight the impact that rare diseases have on families and communities. On Rare Disease Day, millions of patients and families from around the world share their stories to promote awareness of the challenges, hopes, and needs of those living with rare diseases. Learn more

April

Running for Rare

April 6, April 22, and June 15

Every year, the National Organization for Rare Disorders (NORD) raises awareness for rare diseases while fundraising to support undiagnosed patients through their Running for Rare Team. Learn more

June

National Myasthenia Gravis Awareness Month

This event is designed to raise awareness, engage key decision makers and health professionals, and increase funding for research and support programs for caregivers and patients with myasthenia gravis. Learn more

August

Spinal Muscular Atrophy Awareness Month

Families of  SMA was established in 1984; in 2014 they became Cure SMA but contiuned the goal of finding a cure for spinal muscular atrophy.  This event highlights the year-round effort to raise public awareness and increase funding for a cure. Learn more

September

Newborn screening awareness 

The earliest newborn screening occurred in 1967; since then it has become a common practice but more can be done. This event brings together advocacy organizations, states, providers, and families from across the nation in an effort to raise awareness about the importance of expanding newborn screening. Learn more

October

Patient-Centered Care Awareness Month

This global awareness campaign occurs every October and celebrates the progress that has been made in patient-centered care, while highlighting the work that still needs to be accomplished.  It is a time to reflect on the important role that patients and families play in the delivery of healthcare. Learn more

November

National Family Caregivers Month

Celebrated every November, the National Family Caregivers Month is a time to celebrate family caregivers and recognize their importance in healthcare. This event raises awareness of family caregiver issues, educates family caregivers, and increases support for family caregivers. Learn more

December

Infantile Spasms Awareness Week (ISAW) 

This event brings together advocacy organizations, parents, caregivers, decision makers, and healthcare providers in an effort to increase awareness and understanding of infantile spasms, through education and research. Learn more

Disease Awareness and Events Calendar 

Throughout the year, organizations support a variety of initiatives to raise awareness and stimulate conversations about rare diseases. These events bring communities together to help educate the public and influence decision makers in an effort to affect real change in lives of those effect by rare diseases.

February

Rare Disease Day 

February 28

Established in Europe in 2008, Rare Disease Day is now observed in more than 65 countries including the United States Its focus is to highlight the impact that rare diseases have on families and communities. On Rare Disease Day, millions of patients and families from around the world share their stories to promote awareness of the challenges, hopes, and needs of those living with rare diseases. Learn more

April

Running for Rare

April 6, April 22, and June 15

Every year, the National Organization for Rare Disorders (NORD) raises awareness for rare diseases while fundraising to support undiagnosed patients through their Running for Rare Team. Learn more

June

National Myasthenia Gravis Awareness Month

This event is designed to raise awareness, engage key decision makers and health professionals, and increase funding for research and support programs for caregivers and patients with myasthenia gravis. Learn more

August

Spinal Muscular Atrophy Awareness Month

Families of  SMA was established in 1984; in 2014 they became Cure SMA but contiuned the goal of finding a cure for spinal muscular atrophy.  This event highlights the year-round effort to raise public awareness and increase funding for a cure. Learn more

September

Newborn screening awareness 

The earliest newborn screening occurred in 1967; since then it has become a common practice but more can be done. This event brings together advocacy organizations, states, providers, and families from across the nation in an effort to raise awareness about the importance of expanding newborn screening. Learn more

October

Patient-Centered Care Awareness Month

This global awareness campaign occurs every October and celebrates the progress that has been made in patient-centered care, while highlighting the work that still needs to be accomplished.  It is a time to reflect on the important role that patients and families play in the delivery of healthcare. Learn more

November

National Family Caregivers Month

Celebrated every November, the National Family Caregivers Month is a time to celebrate family caregivers and recognize their importance in healthcare. This event raises awareness of family caregiver issues, educates family caregivers, and increases support for family caregivers. Learn more

December

Infantile Spasms Awareness Week (ISAW) 

This event brings together advocacy organizations, parents, caregivers, decision makers, and healthcare providers in an effort to increase awareness and understanding of infantile spasms, through education and research. Learn more

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