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A group of 50 patient-led organizations that are strengthening rare disease communities, improving diagnosis, accelerating research and driving progress in the fight against rare diseases.
Patients are a powerful force in driving research for rare disease — we provide critical insights about our diseases and are fueling significant discoveries.
7,000 rare diseases, one community fighting for cures.
One of our rare mamas, Niki Markou has been featured on the Rare Mamas website that is empowering rare disease moms. Rare Mamas contains blogs, podcasts and resources by rare disease mom founder, Nikki McIntosh who is a writer, speaker and advocate for mamas who care for children with rare disease. “It was an honor to be featured on her page.” said Niki Markou who is one of our Chelsea’s Hope board members
To read the article go to https://raremamas.com/just-like-a-butterfly-by-niki-markou/
October 1st Marks Inaugural Awareness Mission
for Lafora Disease
Lafora Body Disease Day is raising awareness for funding, research, and medical therapies
SACRAMENTO, Calif. – September 30, 2021 – Chelsea’s Hope Lafora Children Research Fund today announced that October 1, 2021, is the inaugural Lafora Body Disease Day. The mission is to create awareness, connect families, fund research, and maintain hope, with awareness efforts continuing throughout the month.
Lafora Disease is a degenerative neurological condition affecting children. Lafora Disease is a genetic condition in which patients are unable to maintain a normal concentration of glycogen, resulting in a toxic accumulation of glycogen, or Lafora Bodies, in the heart, spine and brain. Lafora Disease is terminal, there is no cure. Children first show symptoms in their early teens and typically die within 10 years. Unfortunately, accurate diagnosis is often not made until anti-epileptic medications no longer control the seizures.
“There are no initial signs or symptoms,” said Frank Harris, the President of Chelsea’s Hope Lafora Children Research Fund. “Children are normal, active and intelligent throughout their elementary school years. Seizures are typically the first symptoms, but then the disease shows its true horror. The seizures become increasingly uncontrollable. Then these once normal, healthy children suffer with further physical and neurological degeneration. They lose their ability to walk and talk. They must be fed via a G-tube and need around-the-clock care. And these families know that without a cure, their children will die in a vegetative state, typically within 10 years of onset.”
Families are very involved in raising awareness of Lafora disease, such as Azeza Kasham and Moniqueca Barfield, who have been advocating for medical and financial support in the fight against their children’s affliction. Azeza lost her first-born son, Haitham, at age 16 and is now fighting for her 12-year-old son, Gehad. Moniqueca is a mother of 18-year-old Khari, who developed symptoms and experienced her first seizure when she was 12.
Chelsea’s Hope was created in 2007, when Linda Gerber and a small group of dedicated friends developed a website to share her daughter Chelsea’s story with family and friends. Under their leadership, the organization became the key source of information for affected families and the primary source of research support. It is the mission of Chelsea’s Hope to raise awareness about Lafora Disease, connect with other Lafora families and help fund research to find a cure.
To help fund research to find a cure for Lafora Disease, visit chelseashope.org/donate. Chelsea’s Hope can also be followed on Facebook @chelseashopelaforacure.
Official document PRESS RELEASE_Chelsea’s Hope_Lafora Body disease Day
It’s our first year acknowledging a day for Lafora disease and October 1st is the day.
Lafora Body Disease Day
Our mission is;
Creating Awareness – Connecting Families – Funding Research – Maintaining Hope
Our goal is to raise awareness and much needed funding to continue the research and turn them into therapies. We have done so much in recent years and we are almost there….hope is closer than it ever has been before!
We are dedicating the whole month to creating awareness by asking for help from all of you to ensure we reach our goals!
So please join us for Lafora Body Disease Awareness Month!
Week 1 – Fri 1st October
Message 15 of your friends to set their #AmazonSmile charity as Chelsea’s Hope Lafora Children Research Fund or ask them to donate $20!
Tag us in your posts or hashtag #laforabodydiseaseday #AmazonSmile #EpilepsyFoundation
Week 2 – Fri 8th October
Create an event – Host a special night, invite your friends and ask for a $20 donation.
Tag us in your posts or hashtag #laforabodydiseaseday #laforamovienight or #laforagamesnight or #laforadrinksnight #EpilepsyFoundation
Week 3 – Fri 15th October
“Challenge Your Friends Week”
Do the #OneChipChallenge or #CandyJarChallenge guessing competition and ask for $20 donations or nominate 3 friends to challenge you and pay $20.
Tag us in your posts or hashtag #laforabodydiseaseday #onechipchallenge #candyjarchallenge #EpilepsyFoundation
Week 4– Fri 22nd October
Wear purple lipstick, tag us in your posts and nominate 3 people to donate $20
Hashtag #purplelipsforacure #laforabodydiseaseday #EpilepsyFoundation
Week 5 – Fri 29th October
Create an event – Host a Halloween party, invite your friends and ask for a $20 donation
Tag us in your posts or hashtag #laforabodydiseaseday #laforahalloweennight #EpilepsyFoundation
We look forward to seeing your posts on Facebook
Families are raising awareness to save their children from a fatal genetic condition, Lafora disease.
Moniqueca Barfield from Gardendale, Alabama and Azeza Kasham from Ann Arbor, Michigan are pleaing for support in their fight against Lafora disease. Azeza, mother of three, has already lost her first-born son Haitham to this devastating disease at just 16 years old in 2019 and is now fighting for her second son Gehad, also diagnosed with Lafora disease who is only 12.
“Loosing one son to this disease is enough and now the heartache is harder to bear, going through it all again with Gehad.” – cried Azeza Kasham.
A cure didn’t come in time for Haitham, but there is hope for Gehad. Lafora disease is a single gene disorder. By replacing one gene you can cure the disease. Scientists have shown they can cure the deadly disease in the laboratory. Families like Azeza and Moniqueca are desperate to bring these treatments out of the labs and into the children who need them.
Moniqueca is the mother of 18-year-old Khari McCrary. Khari developed symptoms when she was just 12, and that’s when she experienced her first seizure. Just like all kids with Lafora disease, symptoms start in their early teens and are usually misdiagnosed with milder epilepsy.
It hurts not knowing whether my child will live long enough for a cure, and it hurts seeing her friends in college, starting their young adulthood, and knowing my child will never have that experience. – Moniqueca Barfield
Lafora disease is a terminal neurological degenerative disease, a genetic condition caused by a glycogen storage issue where the body cannot process glycogen, accumulating and resulting in a toxic buildup of Lafora bodies in the heart, spine and the brain.
Once symptoms start, the average lifespan is roughly 2-10 years with most children passing away in the late teens or early twenties.
There are no signs or symptoms initially until the children have symptoms of seizures. Symptoms start in children in their early teens (sometimes earlier) and are usually misdiagnosed with epilepsy. Children are normally quite intelligent and active throughout their schooling years. Symptoms of seizures and cognitive decline progress quite fast, and within the first few years of onset. The doctors realize that something more sinister is wrong when epilepsy medications continue not to control the epilepsy as they should. Seizures become more frequent and uncontrolled over time which results in death. Further testing is done, before getting the devastating diagnosis of Lafora disease.
These children also suffer from difficulty walking and eating, speech difficulty, swallowing, muscle spasms, behavioral changes, cognitive decline, confusion, childhood dementia, photosensitivity, myoclonus and increasingly intractable seizures. These children are constantly in and out of hospital to treat uncontrolled seizures that cannot be contained in the home. The childhood dementia onset also comes on quite quickly, with children becoming disorientated, angry, forgetful, and confused. Watching your parents go through the symptoms of dementia at their old age is heartbreaking, imagine seeing it in your child? “Watching them slowly fade away is gut wrenching.”
Prior to this diagnosis, children like Haitham, Gehad and Khari were like most other teens in the USA, attending school full time with no signs or symptoms of any abnormalities. They were healthy and normal, had social relationships with family, friends, and peers.
Chelsea’s Hope Lafora Children Research Fund have been searching for a cure and creating awareness since 2007. Our mission is to fundraise for research, support the Lafora community, engage key stakeholders and promote the interests of patients. Thanks to the Fund, there have been promising breakthroughs in research, but Covid and lack of funds have caused delays that are taking away precious time. With enough funding we could have clinical trials for children beginning in 2022. So the fight to get the necessary funding to push these forward is what we need now. Time is of the essence for these children and every moment counts.
If you would like to help Moniqueca and Azeza save their children before it’s too late, please go to https://chelseashope.org/donate/ to see ways you can help or email firstname.lastname@example.org.
EnAble Therapeutics has informed Chelsea’s Hope that it is willing to partner with clinicians to lead a n=1 Expanded Access trial or through the Right to Try Act with their potential therapy for Lafora Disease, VAL-1221.
EnAble has indicated that the following medical resources and support will be needed:
Clinicians with institutional support including internal and external regulatory assistance and treatment / I.V. infusion capability.
EnAble has confirmed that this could be your child’s primary neurologist but EnAble has also confirmed that the clinician does not need to be a neurologist.
If interested, please have your clinician contact: