July 29, 2014

Update: Life after ICU

Photo of Bertrand from last Wednesday.  He was happy to be home!
(Yes, the shirt says "chicks love me."  Thanks, Abuelita.)
Bertrand came home from the hospital last Wednesday.  His bedroom obstacle course includes: pulse oximeter, oxygen concentrator, 5 tanks of oxygen, suction machine, and all the tubes, probes, stickers, and doodads that accompany the former.

Despite the accessories, Bertrand is doing very well!  He is down to nighttime oxygen only (but he refuses to leave the nasal cannula in).

Antibiotics are amazing.

Bertrand wasn't the only one on antibiotics.  Victoria also tested positive for mycoplasma, and it is suspected that my 7 week long "cough" was as well.  (Cue the mommy guilt for inadvertently infecting the kiddos.)

Meanwhile, Matthew had big deadlines at work (and a birthday), we've been fielding an avalanche of emails, and oh yeah, we have a 5 week-old baby.

Please forgive us for the delay.

If you have sent an email, I promise we will get to it.  :)  But, it wouldn't hurt to follow-up.

Winston in one of my baby blankets made by Abuelita Elisa.
Yep, it's blue.  Everyone thought I was going to be a boy.  Surprise!

July 23, 2014

Out of the ICU: Ups and down


This is your lung on NGLY1.




Bertrand is into a regular hospital room.


He improved rapidly yesterday (as measured by his ability to breath with decreased assistance), to the point where I got optimistic he might go home.

Unfortunately, his oxygen kept dropping on room air, so they wouldn't release him.

Today, he's still having lower oxygen on room air alone, but it's only problematic when he's asleep.

It's likely he'll be discharged soon, but with a home oxygen machine and portable tanks.

The NIH sleep study results came back, and they showed 10 central apnic episodes per hour (and low oxygen overall), so we'll also be looking at more complete and permanent solutions for his breathing, especially at night.

July 21, 2014

What does community feel like?

The view outside of Bertrand's hospital room: a double rainbow.

The love and support from everyone has been palpable.

With all our heart, thank you.

Rushing Bertrand to the ER and the subsequent PICU admission were scary.

But, we've seen worse.

Bertrand is tricky to diagnose, but doctors managed to identify the cause of his illness quickly. And then to have it be bacterial and treatable? That is all good news.

The most surreal part of this entire episode has been that we had an NGLY1 community/family behind us for the very first time.
  • The Wilseys were the first to know we were headed to the ER--before our own families. 
  • The Leftwich family coached us on exactly what to expect and do with mycoplasma pneumonia and offered support. 
  • The German NGLY1 mom sent funny memes which cheered me up.
  • And, Pam Stinchcomb was my angel, holding my virtual hand through a mini breakdown. (The 19th was Winston's 1 month birthday. Will every one of his birthdays be in the hospital and all about his older brother? Will he resent Bertrand? etc.)

To be clear, we've always felt love and support from our friends in the broader rare disease / special needs / undiagnosed community, but to have people who knew *exactly* what we were going through, and *exactly* what we were feeling? It was transcendent.

I'm overwhelmed with gratitude and joy.

July 20, 2014

In the PICU: Stabilizing

A very happy Bertrand, only days ago.  Photo by Phil Toledano.

Bertrand had a rough night, but I think it was largely because he wasn't allowed to eat.  The concern was that he was still at risk from aspiration during intubation.

During rounds in the morning, I explained that much of his current distress was now due to hunger. (Bertrand's hunger cry is distinct from his pain cry. And, I pointed out that he kept trying to eat the tubes near his mouth.)

I convinced them to start him on a little Pediasure through his nasal-gastric tube, and he stopped crying in minutes.  He's now on a continuous feed and has been relatively peaceful.

In fact, he's slept most of the day, after about 36 hours of being too uncomfortable to rest.

He's awakened every two hours to vacuum out his lungs.  It sounds about like throwing a wetvac in a swamp, and while it's uncomfortable, he clearly feels (and breathes) much better afterward.

The mechanical respirator hums in the background, giving Bertrand's breathing a Vader-like quality.

Bertrand's vitals have steadily improved since this morning.
His heart rate periodically dips into the normal range; his blood pressure is normal; his oxygenation is at 91% with only 40% assistance; and his breathing is much less labored.
The labs from this morning showed that the infection is not worsening, and it's expected to improve steadily from today.
Our hope is that he'll improve enough to be transferred to a regular room tomorrow.

July 19, 2014

Off to the ER: Mycoplasma (bacterial) pneumonia


Bertrand's streak had to end at some point.

Bertrand hasn't been hospitalized (for illness) in over two years -- since about the same time as his diagnosis as NGLY1 deficient.

But, we're back.

Bertrand has had a mild cough for about a week.  We've all had it to some degree.

Bertrand doesn't get sick often, but when he does, he usually recovers normally and without assistance.

As in the past, Bertrand seemed to be stable or mending.

Last night, his symptoms worsened precipitously.

He was crying, coughing and refusing liquids (but not solids).

Cristina and I were up all night on and off tending to him and Winston.

Shortly after his breakfast, Bertrand's eyes, nose and lips started turning bluish purple, he began crying unconsolably and his body was racked by tremors.

We couldn't tell if it was a seizure or an aggravation of his movement disorder, but whatever it was, it was definitely new.

We tried to get a reading on his oxygen with his a pulse oximeter, but he was shaking so violently that we couldn't properly attach the lead.

So, we piled into the car and drove to the ER.  (We very deliberately live only a minute a way from the pediatric ER.)

I carried Bertrand through the door, and after looking at him for three seconds, the admissions specialist escorted us directly to an open trauma room.

A platoon of twelve docs, nurses and specialists descended on Bertrand.

Given his purplish discoloration, he was placed on manual ventilation instantly.

Seconds later two IVs were in.  (I still don't know why they did two.)

Specialists and nurses started attaching leads and tubes so fast that I couldn't track what was being done to him.

A few minutes after entry, Bertrand looked like the Borg again.

Heart rate was tachycardic and rising.  Body temperature was 102 F.  Blood pressure was low and plummeting.  Oxygenation was in the 80% range.  Breathing was painful and labored.  They kept referring to poor perfusion from his mottled skin.

A single tear rolled out of Bertrand's right eye.

We began trying to explain Bertrand's medical history to the resident and the physician, sorted by most to least relevant information for the evolving emergency.

As Bertrand's breathing worsened, the physician pulled us out of the room to speak with us in private.

"Since he has a serious genetic condition and it looks like he may need a breathing tube shortly, I want to know if you would like to allow the assisted respiration.  I apologize for asking, but with these sorts of conditions, I have to check."

Cristina and I had been asked to consider putting "do not resuscitate / do not ventilate" directives in Bertrand's medical file years earlier.

We didn't even have to look at each other before responding: "Intubate him. If necessary. Whatever it takes."

Cristina added defensively, "He's normally very happy. He has a great quality of life."

The attending ER physician felt he was having a seizure (as did we), so she gave him Ativan (Lorazepam) as a rescue medication.

The tremors subsided momentarily.

Even as the IV fluids went in, his blood pressure continued to drop.

They gave him more fluids.  No effect.

As his blood pressure neared critically low levels, the physician put him on dopamine.

It worked.  His blood pressure started rising.

The manual respiration had his oxygenation back up to 99%: his lips, nose and eyes were pink again.

His heart rate stayed elevated, but no longer dangerously so.

The tremors continued, but it seemed like the rest of Bertrand was stabilizing.

The tension in the room visibly eased.

The phlebotomy team collected blood from his feet.  A catheter went in.

After about an hour, Bertrand had passed out, so they transferred us to the pediatric intensive care unit (PICU).

Waiting for the elevator, no one spoke.  For first time since we walked in the door, we had silence.

Up in the PICU, they informed us that Winston would have to leave, since children were not allowed.

Under significant protest, Cristina left me in charge.

About half an hour after getting into the PICU, the resident told the nurse, "Mycoplasma pneumoniae."

I jumped in: "That's bacterial, right?"  (I knew it was bacterial, because I'd looked it up three weeks earlier when I found out that another NGLY1 patient had had it.)

They confirmed and said that ordinary antibiotics would not work, since mycoplasma lack the cell wall targeted by many antibiotics.

Cristina was googling at home and had already discovered that they were likely to recommend erythromycin (or a variant).

Unfortunately, that induced (potentially fatal) long QT syndrome in Bertrand's heart about four years ago.

When they came back, they said they were going to start azithromycin (a variant on erythromycin).  I explained the issue with his heart and long QT.

After consulting with pharmacist and Cristina by text, we went with azithromycin (over worse options), but under careful cardiac observation for five days, with a baseline EKG taken immediately.

While waiting for the azithromycin, a respiratory team suctioned out his lungs, and they were able to lower his oxygen a little after that.

Bertrand is now relatively stable and on pain medication, but they're withholding food until his lungs improve.

He's hungry and uncomfortable, but he looks a lot better than he did this morning.

I'm optimistic that Bertrand will respond quickly to the antibiotics, but it's going to be a long five days for him.

I would hardly call us complacent in the search for a treatment.

But, this and recent events with other NGLY1 patients are a reminder of just how fragile these kids are and of the urgency of finding a viable treatment.

Once Bertrand is well and home, we'll resume the hunt with haste.

July 18, 2014

NGLY1.org Facebook Launch

Yesterday, we launched the NGLY1.org page on Facebook to facilitate N-glycanase (NGLY1) deficiency family-researcher interaction and provide support.

We invite everyone to join.  The page will be a public one, but we reserve the right in the future to make it private if necessary.

In 2012, we launched NGLY1.org as a resource for clinicians, a means of finding new N-glycanase deficiency patients, and a way to support their families.

We will continue to promote N-glycanase deficiency, so patient families will be able to find our community and receive the same support we find so valuable.

We are no longer alone.  :)

July 17, 2014

Bertrand's story hits The New Yorker!

Our family is grateful to journalist, bestselling author and MIT professor, Seth Mnookin.  His time, thoroughness, and dedication to the NGLY1 story were astounding.  We are also indebted to The New Yorker.  Their staff of editors and fact checkers left us awed.  This was a work of true, old-school journalism.  We're proud to be a part of it.

ONE OF A KIND

What do you do if your child has a condition that is new to science?

BY SETH MNOOKIN
JULY 21, 2014

Until recently, Bertrand Might was the only known patient with a certain genetic disorder. His parents began searching for others. Photograph by Phillip Toledano.
Matt Might and Cristina Casanova met in the spring of 2002, as twenty-year-old undergraduates at the Georgia Institute of Technology. Cristina was an industrial-design major with an interest in philosophy; Matt was a shy computer geek obsessed with “Star Trek.” At first, Cristina took no notice of him, but the two soon became friends, and that fall they began dating. Within a year, they were married.
The couple had their first child, a son, on December 9, 2007, not long after Matt completed his Ph.D. in computer science and Cristina earned her M.B.A. They named him Bertrand, in honor of the British philosopher and mathematician Bertrand Russell. After a few blissful weeks, the new parents began to worry. Matt and Cristina described Bertrand to friends as being “jiggly”; his body appeared always to be in motion, as if he were lying on a bed of Jell-O. He also seemed to be in near-constant distress, and Matt’s efforts to comfort him “just enraged him,” Matt says. “I felt like a failure as a father.” When the Mights raised their concerns with Bertrand’s doctor, they were assured that his development was within normal variations. Not until Bertrand’s six-month checkup did his pediatrician agree that there was cause for concern.
By then, Matt had a new job, as an assistant professor at the University of Utah’s School of Computing. It took two months to get Bertrand on the schedule of a developmental specialist in Salt Lake City, and the first available appointment fell on the same day as a mandatory faculty retreat. That afternoon, when Matt was able to check his phone, he saw that Cristina had left several messages. “I didn’t listen to them,” he told me in an e-mail. “I didn’t have to. The number of them told me this was really bad.”  
Read the rest HERE.

July 15, 2014

Photo Outtakes


The Rule of 3: When taking photographs of three children, at most two will respond appropriately.  ...and the other(s) will be staring off distractedly or screaming.












I love these photos.  
They capture the perfectly imperfect, happy, craziness that is us. 

July 12, 2014

Baby Winston


These photos were taken by Staci (Sweet Envy Photography) when Winston was 6 days-old.
Everything about our little man is a joy!  He's the perfect eater, sleeper, cuddler, and giggler.






July 10, 2014

3rd Annual RARE Patient Advocacy Summit

September 11-12, 2014
Hyatt Regency Resort & Spa, Huntington Beach, California


Register today!

 
What if you could learn EVERYTHING you needed to know about being a patient advocate in just two days? What if you could learn how to be prepared, proactive, and productive in your efforts to better the lives of those with rare disease? What if you could be an advocate with the know-how to lobby congress and effect change? What if you felt you really had the ability to reach out to the largest pharmaceutical companies in the world—and form a powerful relationship to bring drugs to patients now?
 

This is what over 200 participants will be learning in-person, and over 5,000 via Livestream during the Global Gene’s 2014 RARE Patient Advocacy SummitSeptember 11-12, at the Hyatt Regency Resort & Spa in beautiful Huntington Beach, CA.
 
This year, participants can expect more toolsexperts, and exploration than ever before. Everything has changed. The feedback we received from the last few years has been positive, but what we now understand is how much more depth our community wants and needs on topics related to their rare disease journey—and we’re determined and ready to give them that!

This year’s sessions, lead by an extraordinary team of experts, include modules on:
  • Caregiving: Strategies for Staying Afloat, Presented byCaregiver Action Network
  • The E-Patient Revolution, Presented by Health 2.0 andSmart Patients
  • Patient-Centered Benefit-Risk Assessment, Presented byFasterCures
  • Making your organization an “Unstoppable Charity”
  • Lobbying at the State and Federal Level
  • Transition & Transformation with Rare Disease from Adolescence to Adulthood.
  • Must Have Collaborations for Successful Drug Development

Also NEW this year:
  • Deep Dives - branched-off discussions that will allow small groups to interact with subject matter experts to help them go more deeply into the content of each session have been added to the agenda.
  • Science Briefs -15 minute science pitches with 5 minutes of Q&A, discussing some of the most promisingInnovations in Science.

Can’t attend in person? Our new Livestream component will allow up to 5,000 advocates from around the globe to attend virtually via webcast at no cost! The event will be broadcast live with opportunities for patients to participate from afar using social media such as twitter and Facebook, using the hashtag #2014GGSummit.

 
Read more about the RARE Patient Advocacy Summit here.

To register for this event (in-person or via Livestream), please visit: http://globalgenes.org/events

July 9, 2014

2014 Special Needs Fine Arts Camp



Fine Arts Camps for
Children & Young Adults with Special Needs
July 14-18, 2014

It isn’t too late to register for this exciting camp experience.
This week-long camp is only $35, but there are a few spots remaining in each camp.

Tanner Dance at the University of Utah is pleased to offer fine arts camps for children with special needs, focusing on dance, music, theater, and visual arts. Please call the office at 801.581.7374 or register online. Please share this camp information with anyone who may be interested.


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These unique fine arts half-day camps for children and young adults with special needs will include dance, music, theatre, and visual arts. With our positive approach and engaging philosophy, this experience will be different from any other. The faculty are trained to work with children of all abilities.

Class
Day
Time
Tuition
Register
Ages 5-10
M-F
9:15 a.m.-12:15 p.m.
$35
Ages 11-18
M-F
1:15 - 4:15 p.m.
$35



Tanner Dance is located in the beautiful new Beverley Taylor Sorenson Arts and Education Complex on the University of Utah campus.  For questions about the camp, please call (801) 581-7374.

July 8, 2014

Utah Hemp Extract Registrant #1


This morning, Bertrand became the first person in the state of Utah to receive a Hemp Extract Registry Card.


While Bertrand may not receive CBD oil for quite some time, we wanted to show our support for the new law on the very first day.  

The only time I could fit a trip down to the Utah Department of Health was at 9:45am, after school bus pick-up, camp drop-off and some errands, but before school bus drop-off, camp pick-up, and a doctor's appointment.  (And, let's not forget, nursing baby.)

Since the Office of Vital Records opens at 8AM, we did not expect to be the first in line.


There was a bit of confusion from some of the staff when we first arrived, but things were quickly straightened out.  

Since all of our paperwork was in order ahead of time, getting the card only took 30 minutes (and should be faster for subsequent registrants).

The Hemp Registry Instructions on the Utah Department of Health website were very thorough and easy to follow.


The Department of Health is accepting applications via mail or in-person.  
For the first week only, walk-ins are welcome, but appointments will be required in the future.
Please see the Utah Department of Health website for more information.


I broke my no-selfie rule to show Bertrand one of the kind registrars at the Dept. of Health, Leisa Finch, who is helping kids like him get access to these cards.  

A big hip hip hooray for the state of Utah, our representatives and senators, and the fantastic folks at Hope 4 Children with Epilepsy who made this all possible!

July 6, 2014

The brothers work out together.


Bertrand loves playing with this new ball from his German best friend! Meanwhile, baby Winston listens to the laughter and does some tummy time.  These are some hardworking brothers.  ;)

July 1, 2014

NIH names new clinical sites in Undiagnosed Diseases Network



David Goldstein and Vandana Shashi, who headed up the amazing team at Duke that discovered N-glycanase deficiency, will also be heading up one of the sites for the Undiagnosed Diseases Network (UDN)!  The UDN is a large step forward for undiagnosed patients everywhere.  We are hopeful that many families will soon be getting answers thanks to fantastic researchers like Drs. Goldstein and Shashi.
Bethesda, Md., Tues., July 1, 2014 - The National Institutes of Health has awarded grants to six medical centers around the country to select from the most difficult-to-solve medical cases and together develop effective approaches to diagnose them. The clinical sites will conduct clinical evaluation and scientific investigation in cases that involve patients with prolonged undiagnosed conditions. Each clinical site will contribute local medical expertise to the NIH Undiagnosed Diseases Network (UDN). The network includes and is modeled after an NIH pilot program that has enrolled people with intractable medical conditions from nearly every state, the District of Columbia and seven foreign countries. The network builds on a program at the NIH Clinical Center in Bethesda, Md., that for the past six years has evaluated hundreds of patients and provided many diagnoses, often using genomic approaches, for rare conditions. 
"Newly developed methods for genome sequencing now provide us amazingly powerful approaches for deciphering the causes of rare undiagnosed conditions," said Eric D. Green, M.D., Ph.D., director of the National Human Genome Research Institute. "Along with robust clinical evaluations, genomics will play a central role in the UDN's mission." Dr. Green and Story Landis, Ph.D., director of the National Institute of Neurological Diseases and Stroke, co-chair the UDN working group. 
Undiagnosed diseases are conditions that even skilled physicians cannot diagnose despite extensive clinical investigation. They may not be recognized by doctors because they are rarely seen, are previously undescribed, or are rare forms of more common diseases. 
The NIH Common Fund awarded four-year grants of approximately $7.2 million (pending available funds) to each of the six medical centers around the country. James M. Anderson, M.D., Ph.D., director of the NIH Division of Program Coordination, Planning, and Strategic Initiatives (DPCPSI), announced in an NIH telebriefing that the six newly awarded sites join a clinical site already established at NIH in pursuing cutting-edge diagnoses. In addition, this past December, NIH selected Harvard Medical School as the UDN Coordinating Center for the multi-institution network. "The NIH Undiagnosed Diseases Network has the potential to transform medicine and serve as a catalyst for new discoveries," said Dr. Anderson. "It is an ideal NIH Common Fund program-the only one focused on diagnoses of rare disorders." 
The following institutions were awarded grants to establish UDN clinical sites:
  • Baylor College of Medicine, Houston; Principal Investigator: Brendan H.L. Lee, M.D., Ph.D.
  • Boston Children's HospitalBrigham and Women's Hospital, and Massachusetts General Hospital, Boston; Principal Investigator: Joseph Loscalzo, M.D., Ph.D.
  • Duke University, Durham, North Carolina; Principal Investigators: David B. Goldstein, Ph.D, and Vandana Shashi, M.D.
  • Stanford University, Stanford, California; Principal Investigators: Euan A. Ashley, M.D., D.Phil., Jonathan Bernstein, M.D., Ph.D., and Paul Graham Fisher, M.D.
  • University of California, Los Angeles; Principal Investigators: Katrina M. Dipple, M.D., Ph.D., Stanley Nelson, M.D., Eric J. Vilain, M.D., Ph.D., and Christina Palmer, C.G.C., Ph.D.
  • Vanderbilt University Medical Center, Nashville; Principal Investigators: John H. Newman, M.D., and John A. Phillips, III, M.D.   
"This type of program can invigorate a medical center anywhere in the country and in the world," said William A. Gahl, M.D., Ph.D., clinical director at the National Human Genome Research Institute (NHGRI), director of the NIH-based Undiagnosed Diseases Program (UDP) and co-coordinator of the UDN working group. "Often, patients have a lot of physical complaints and no objective diagnoses. Our goal is to use the latest tools to make a diagnosis that spans the clinical, pathological and biochemical spectrum to uncover the basic genetic defect." Since 2008, the UDP has explored this fascinating area of medical research and acquired practical insights in the process of enrolling approximately 600 undiagnosed children and adults in its clinical protocols. The multidisciplinary clinical and research team diagnosed approximately 100 patients (20-25 percent of those evaluated), discovered two unknown diseases and identified 15 genes not previously associated with any other human disease. A combination of genomic and clinical analyses contributed to the diagnoses. 
By including an additional six clinical sites, the NIH UDN will both draw upon the unique expertise of new clinical research groups and cultivate opportunities for collaboration among a larger group of expert laboratory and clinical investigators. Physicians within the network will collect and share high-quality clinical and laboratory data, including genomic information, clinical observations and documentation of environmental exposures. They also will benefit from common protocols designed to improve the level of diagnosis and care for patients with undiagnosed diseases. 
 "The UDN will look at diseases across all clinical specialties using new tools and methods of analysis for the identification of new diseases," said Anastasia L. Wise, Ph.D., a program director in NHGRI's Division of Genomic Medicine and co-coordinator of the UDN working group that oversees the development and implementation of the UDN. "The network will facilitate collaboration and shared use of genomic tools among the sites." Based on the NIH UDP experience, the UDN Coordinating Center at Harvard Medical School has begun paving the way for the new UDN clinical sites to begin accepting patients. Among the coordinating efforts are the preparation of draft protocols and operating guidelines, and the definition of an initial framework of common practices across the network. The network will share systems for data collection and develop common approaches to patient selection, evaluation and diagnosis. 
Each new clinical site may have variations in handling health insurance coverage for clinical testing and care. However, no patient will be turned away from participation in the UDN based on lack of health insurance coverage. 
"We believe that there is a substantial unmet demand for the diagnoses of conditions that have perplexed skillful physicians," said Isaac Kohane, M.D., Ph.D., professor of pediatrics at Harvard Medical School and Boston Children's Hospital and principal investigator of the Coordinating Center. "We want to address inquiries from physicians and patients throughout the country who require these services and, in doing so, create a 21st century model for diagnosis and treatment in this genomic and information-intensive era." 
UDN investigators will share genomic data from UDN patients with the research community through multiple public repositories. Network-wide data sharing will observe standards of patient privacy, confidentiality and management of health information.  
The network will start up and test its operating procedures during its first year. It will progressively expand recruitment of patients so that by the summer of 2017, the rate of admissions at each new clinical site will be about 50 patients per year. For a period this summer, referrals from clinicians on behalf of undiagnosed patients may continue to be made through the existing NIH application pipeline. 
Instructions on applying to the UDN on behalf of a patient can be found at rarediseases.info.nih.gov/undiagnosed.
For more information about the UDN, including related funding announcements, visit http://commonfund.nih.gov/Diseases/index.
These UDN clinical site awards are supported by NIH grants 1-U01HG007672-01, 1-U01HG007674-01, 1-U01HG007709-01, 1-U01HG007690-01, 1-U01HG007708-01, 1-U01HG007703-01. 
NHGRI is one of the 27 institutes and centers at the National Institutes of Health. The NHGRI Extramural Research Program supports grants for research and training and career development at sites nationwide. Additional information about NHGRI can be found at www.genome.gov.
The NIH Common Fund encourages collaboration and supports a series of exceptionally high-impact, trans-NIH programs. Common Fund programs are designed to pursue major opportunities and gaps in biomedical research that no single NIH Institute could tackle alone, but that the agency as a whole can address to make the biggest impact possible on the progress of medical research. Additional information about the NIH Common Fund can be found athttp://commonfund.nih.gov.
National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 institutes and centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.