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| Jonah's last day of 5th grade. |
If only I had a secret tunnel from my apartment to my office
so that I didn’t have to interact with anyone. This morning on my walk to work,
I’m waiting at the intersection, an ambulance is trying to get down the side
street. There are a dozen cars that have nowhere to go, the streets aren’t wide
enough for them to go anywhere. The car at the front of the line is trying to
park. She backed in and out, multiple times, meanwhile the other cars are
blaring their horns and waiving at her to go. The ambulance is bearing down on
everyone and she’s taking her sweet ass time to park. Because you know parking
spots in Brooklyn are priceless. I walk up to the car and yell to the women, “Do
you not hear the ambulance behind you?” She gave me a put off look and pulled
into the intersection, letting everyone go. Her precious parking spot is more important
than someone’s life.
There are those moments where I just can’t keep my damn
mouth shut. For instance, earlier this week I yelled at a guy for throwing his
garbage out the window. He laughed at me. Later that day I picked a fight with
a man that someone like me should avoid at all costs. I wouldn’t feel so bad
about it if I had not had Jeremy and Jonah with me. I may or may not come back
to this story, I’m ashamed of what I did.
My anger, started with politics and red tape with the NIH
and this current grant. I came very close to having to shut Phoenix Nest down
because of the freeze placed on our grant. We had a primary investigator change
on the grant that threw the NIH off, they were not happy. With just cause, this
is one of their larger grants. I assume that it was controversial in that it
went to a virtual biotech led by a mother with a BS in fashion and a BS in
business management. The NIH can’t be biased against me personally, PN won the
grant fair and square, the reviewers gave us an almost perfect score, the
science and path forward was sound and rational. The NIH froze our grant until
I could find a suitable PI replacement, I found our replacement a few months
ago. Yet, the grant stayed frozen while the NIH found time to make new
milestones for us, this is still in play. After begging they released enough
funds for me to pay salaries, OH and subawards, through June. I anticipate that we will be back on track in the next week. This has been
going on since February 4th.
The NIH does not have the staffing needs to see their
programs through in a timely and seamless fashion. I don’t blame the NIH; I
blame the system. I blame the people that put this system in play. I blame my
friends, family and neighbors that don’t pay attention to what their choices
are doing to their friend’s neighbors and family members. Everyone over 18 has
the opportunity to vote.
The Hearing on: “Reauthorizing Vital Health Programs for American Families” https://youtu.be/_gG9T8tKzqA Really got to me and it should get to you too.
These four proposed bills will affect every single one of us.
H.R.
776, the "Emergency Medical Services for Children Program
Reauthorization Act of 2019"H.R. 1058, the "Autism CARES Act of 2019"
H.R. 2035, the "Lifespan Respite Care Reauthorization Act of 2019"
H.R. 2507, the "Newborn Screening Saves Lives Reauthorization Act of 2019"
If you don’t think a rare disease or Autism will play a
factor in your life and NBS isn’t a priority for you. Then turn your attention
to H.R. 2035, we are all going to get old and many of us will get cancer, Alzheimer’s
or some other debilitating, expensive and painful medical condition. Who’s
going to take care of you? If you don’t pay attention it won’t be the
government, your tax payer dollars are going towards the department of defense.
Your insurance might cover surgeries, meds and other treatments. But who’s
going to feed you your apple sauce and meds? Who’s going to wipe your butt,
read to you and put you to bed?
The President’s Fiscal Year (FY) 2019 Budget supports the
mission of the Department of Health and Human Services (HHS) by making
thoughtful and strategic investments to protect the health and well-being of
the American people, address the opioid crisis, promote patient-centered health
care, strengthen services for American Indians and Alaska Natives, encourage
innovation in America’s health care future, address drug pricing, advance
regulatory reform, and focus resources toward proven and effective initiatives.
The President’s Budget request for HHS proposes $95.4 billion in discretionary budget authority
and $1,120 billion in mandatory funding to carry out the mission of the
Department and to fulfill the promises made by the Administration to the
American people.
President Donald J. Trump signed a $1.3 trillion spending
bill on March 23, 2018 that includes a $160 billion boost in defense spending over two years,
reversing years of decline and unpredictable funding.
95 billion is about half of 1.3 trillion. That’s what Siri said,
I can’t count that high.
Both the DOD (The Department of Defense) and HHS (Department
of Health and Human Services) fund numerous other programs related to their department
agenda. For instance, the Department of Health and Human Services’ funds the
NIH and the FDA. While the DOD funds the CIA and FBI.
Everyone knows what the CIA and FBI does, made popular by
blockbuster movies and Trump’s antics. The FDA and NIH Food and Drug
Administration and the National Institute of Health, on the other hand, not so
sexy. You hear FDA and you think E. coli in your lettuce or measles outbreak at
the school in the town 60 miles from your town and think phew, it’s not my town
and I don’t eat lettuce, I’m safe. The
NIH… I’m surprised by the number of people that don’t know what the NIH is or
does. The NIH saves lives. The NIH helps create treatments that will cure
diseases forever and ever. You sense the anger here?
My point is to scare those that don’t realize that their VOTE
matters not just to you but your entire family. For those that don’t have a
pension, social security or savings If you get cancer and don’t have the cost
to cover your care then it falls on your family.
I assume that a major argument would be that people believe
that immigrants eat up our health care so that they should be shut out of the
United States. This is the opposite of the truth. https://www.modernhealthcare.com/article/20180808/NEWS/180809934/study-finds-immigrants-use-fewer-u-s-healthcare-resources
I'm reading out loud to Mari, she's telling me to stop that I'm making myself crazy. I know I am. 'Besides, she says all the guys in the red hats are just going to say it's fake news.'
Personally, for my family and my MPS community we struggle
on a daily basis for the care of our children. Jeremy and I fear what will
happen to Jonah if he out lives us, who will provide for him? Imagine having three
adult MPS children all of whom present as an elderly person with serious mental
and health issues, all need full time care. You never get a break! Respite
federal funding is almost non-existent. Since 2009 when this program was enacted
only 37 states received at least one federal assistance grant, which equaled
less than a million dollars a state. Last I counted there were 50 states, 10
years this program has been in existence and it’s still not benefitting 85% of
the people that need help. It’s heartbreaking, some families decide not to use
the services because the providers are so poorly trained that it’s unsafe to
leave their loved one in the providers care. Jeremy reminded me of the jury
duty service that I presided on years ago. A felony murder, two young women
from a respite service provider attacked their client, an elderly holocaust
survivor. They wanted her social security check, they didn’t get it, they found
$27.00, they tied the women up and left her for dead.
The public-school system for special needs children
sucks beyond belief. You can’t even imagine the lack of placement for our kids.
In NY state you have to sue the DOE to place your child in a private school. We
started that process years ago, spent a few thousand in the process, having to
have Jonah evaluated out of our own pocket with a private neuropsychologist.
Jeremy and I started touring schools, there are some good private schools out
there, but you have to fight to get a placement it can take years. The schools
we liked were an hour and a half bus ride each way. If Jonah had a meltdown,
pooped his pants or got sick he’d half to wait hours before I could get out to
him. Some private schools cater to severe behavioral issues, others to
non-verbal autistic kids. We never found a good fit.
Fortunately, our public school had a place for him in their 12:1 class, we have been pretty lucky up until now. Moving into middle school, even typical kids have to apply to schools. For Jonah there was only one choice. We got in… so we thought. It’s the last week of school and we’re told that Jonah wouldn’t be a good fit in the 12:1 class, since the children in that class we not make good peers for him, they fought with each other. Great…. And we hear this through Jonah’s current teacher not the school that accepted him.
We might be able to get him into their ACES program, but we were supposed to have him take an ‘alternative evaluation’ test at the beginning of the year, it’s too late for them to open his IEP now. We applied anyways. I called the principals at both schools and the psychologists at both schools. Each person has a different answer.
The principal at the new school promises me that Jonah will get into her ACES program. My principal, says "no they made a mistake, Jonah’s current IEP depicts that he’s not qualified."
I finally get the email address of the person that makes the final decision on who gets placed in the handful of ACES programs in NY. You can’t find this person’s name or # anywhere online, the ACES programs are a virtual secret. They’re designed to help children with moderate autism, downs syndrome etc. learn life skills. A perfect spot for Jonah and we’re only now hearing of the program.
The ACES director calls me and states: “Jonah does not have a spot into the ACES program until I say he has a spot.”
She goes on to say, "I review the applications in which they were received, Jonah’s application was just sent in. “Furthermore, these spots are highly coveted, and she has a stack of applications.”
Fortunately, our public school had a place for him in their 12:1 class, we have been pretty lucky up until now. Moving into middle school, even typical kids have to apply to schools. For Jonah there was only one choice. We got in… so we thought. It’s the last week of school and we’re told that Jonah wouldn’t be a good fit in the 12:1 class, since the children in that class we not make good peers for him, they fought with each other. Great…. And we hear this through Jonah’s current teacher not the school that accepted him.
We might be able to get him into their ACES program, but we were supposed to have him take an ‘alternative evaluation’ test at the beginning of the year, it’s too late for them to open his IEP now. We applied anyways. I called the principals at both schools and the psychologists at both schools. Each person has a different answer.
The principal at the new school promises me that Jonah will get into her ACES program. My principal, says "no they made a mistake, Jonah’s current IEP depicts that he’s not qualified."
I finally get the email address of the person that makes the final decision on who gets placed in the handful of ACES programs in NY. You can’t find this person’s name or # anywhere online, the ACES programs are a virtual secret. They’re designed to help children with moderate autism, downs syndrome etc. learn life skills. A perfect spot for Jonah and we’re only now hearing of the program.
The ACES director calls me and states: “Jonah does not have a spot into the ACES program until I say he has a spot.”
She goes on to say, "I review the applications in which they were received, Jonah’s application was just sent in. “Furthermore, these spots are highly coveted, and she has a stack of applications.”
Jonah’s graduation was beautiful. The teachers gave him an
award for excellence in art and design. Jonah was so happy to run up on stage
and retrieve his award. It was and always will be a very special moment for Jeremy
and I. Only one student out of the whole 5th grade received that award,
I’m sure it could have gone to a child with exceptional skills. But they gave
it to Jonah, who is exceptionally talented in the nontraditional way.
 |
| So proud! Jonah gives the thumbs up. |
Did you see this clip? https://themighty.com/2019/06/indiana-autistic-student-most-annoying-award/?fbclid=IwAR3ubvMyL0IUlclCZprbL7k0w-F1Z3NG7d3dqAMOhI2bS6BRvZAa9-uZB7Q
I was so outraged I called the super
attendant of schools and demanded that all the teachers involved be fired and
that Autism sensitivity be taught to the entire states school district. I wasn’t
the only one who called, the school administrators took swift action, three
teachers were immediately fired, the school principal is under investigation
and they are implementing sensitivity training. YES! I’m so happy that people from
across the country took the time to call and voice their anger. We can make
this country great again, if we band together, to help one another.
Finally, I have been extremely discouraged on my ability to find a
sponsor to commercialize the two drugs that we have licensed, gene therapy for
IIIC and ERT for IIID. Not my ability really but the reality that we are too
rare. Another large drug company has announced that they are trying to sell
their MPS III programs that is currently in trial. If they can’t find a buyer,
the drugs will just go away.
Recently a gene therapy drug was approved for SMA, a horrible
pediatric disease, I was perked up by this news. There was a huge amount of
publicity behind this drug, it is the most expensive approved drug in the world.
There are people out there who don’t think a rare disease is worth the price. Then I read the patient incident of this rare
disease I in 53,000. Jonah’s disease is 1 in 1.5 million.
People don’t pray for me vote for me, vote for your family,
friends and neighbors. Pay attention to where your tax payer dollars are going.
Drones vs Humanity?
On my birthday, a newly diagnosed dad called me, he was in
his car and he asked, “can I drive to your office and just sit there?”
He ended up spending two nights. I gave him a crash course
in everything Sanfilippo IIIC. He hasn’t come out of the closet yet, so didn’t post
any pictures of my Birthday, because he’s in them.
His son is two weeks older than Jonah and has the same
subtype. He had been living under an Autism diagnosis since he was 5, that’s 6
years. One day this man’s client mentioned to him that his son was genetically tested,
and they found that he had a rare disease. This sparked something in this dad,
and he decided to ask his doctors to give his son another genetic test. Thinking
maybe the science had evolved and maybe they might find something new. It did.
At my house, the dad sat with Jonah playing and reading to
him. He was in disbelief of how similar
our boys are. I knew it would flip him out. Our boys’ mannerisms, questions,
likes, and facial features virtually mirror each other. I asked him if he had
seen Jonah in an interview would he realize that his son had the same disease? He responded: “without a doubt”
We need more press, I know these children are out there,
just like this boy they have been dx’d as autistic.
I fear that I won’t be able to convince a partner to
commercialize our drug that we fought so hard to fund.
I attempt to bury my fears in work, reminding myself that so
many people are helping and cheering for us. The Cure Sanfilippo Foundation have
been very supportive and have helped restore my faith. Thanks Cara and Glen.
Now that I got this off my chest, I feel better. Going to
bed now. Tomorrow is another day.
