Only you can SAVE me!

Hi, everyone!

I just created community to search for charitable donations for my spine surgery and my medical examination before surgery.
I look forward to your help, because my condition is getting worse all the time.

And only you can SAVE me.

My community: https://www.facebook.com/groups/needhelpmonika/

I need you help!

I ask for your help!

My name is Monika Lemeshonok, I am 30 years old, I am from Belarus. From born I have disease Spinal Muscular Atrophy 2 type, I can’t walk and move. From my disease I have weak back muscles and it is don’t support of the spine, from that I have a strong scoliosis, I can’t sit without support and crushed the lungs, breathing heavily, I have back pain every day. I needed surgery scoliosis, which will help to correct my position and to improve my health; I will be able to sit without help herself. This surgery on the spine for people with my diagnosis made in the Instituto Ortopedico Rizzoli, Italy (Bologna). I sent an inquiry to this clinic and they are ready to take me to a medical examination before surgery in September. After medical examination in clinic will composed of the cost of quota to hold my surgery.

The donation funds will be used to pay of my visit to Italy, for medical examination and consultation with doctors, surgery and 1 month of rehabilitation in the Instituto Ortopedico Rizzoli of Bologna (clinic will give a more detailed estimate after my visiting).

Thanks so much! More info: https://www.facebook.com/events/160187677729377/


You can send needed money to:

USD

1. USA
CITI BANK N.A., NEW YORK
SWIFT code: CITIUS33
ACCOUNT NUMBER: 36316365

Belarusbank
WIFT-code: AKBBBY2X
BIC 153001795 (code 795)
Payer’s Identification Number: UNP 100325912
Belarusbank
Mogilev Regional Branch 700
BIC код АКВВВY 21700
For transfer on account № 000005 in branch №700/70
Belarusbank , city Mogilev code 536

— PURPOSE OF PAYMENT — ,
Name of client: Lemeshonak Monika


PayPal: Lemesonok@gmail.com

VISA Electron: 4255 2000 5198 3740

SMN Protein Can Be Measured in Blood – Implications for Clinical Trials

The majority of therapeutic approaches currently in clinical development for SMA, aim to increase the levels of the SMN protein, either through the back-up gene, SMN2, or through the replacement of the missing gene, SMN1. There is therefore a need for sensitive methods to quantify increases in SMN protein in blood and other accessible tissues.

Phillip Zaworski of PharmOptima LLC and Katharine von Herrmann of the SMA Foundation, along with their team, have found a new method, an assay, to detect levels of SMN protein in the blood. Using the assay, they measured SMN protein in whole blood from SMA patients and healthy controls and found that SMN protein levels were associated with SMN2 copy number and were greater in SMA patients with 4 copies, relative to those with 2 and 3 copies. SMN protein levels did not vary significantly in healthy individuals over a four-week period and were not affected by circadian rhythms. Almost half of the SMN protein was found in platelets, components of the blood involved with clotting and healing. The team also showed that SMN protein levels in mice with a mild form of SMA, were high in the neonatal stage, decreased in the first few weeks after birth and then remained stable throughout the adult stage. Importantly, SMN protein levels in the CNS correlated with SMN levels measured in whole blood of these mice. These findings have implications for the measurement of SMN protein induction in whole blood in response to SMN-upregulating therapy.

http://www.smatrust.org/smn-protein-in-blood-can-be-measured-implications-for-clinical-trials/

Summer warm evening

Summer warm evening

I'm out for a walk in a city park.

Biogen and Ionis Release Spring 2016 Community Update

Biogen and Ionis Pharmaceuticals recently provided an update on the clinical development of nusinersen (IONIS-SMNRx), which is currently being tested in Phase 3 clinical trials:

As we approach the upcoming Cure SMA conference in Anaheim, we want to provide an update on the status of the nusinersen program and its direction.

Enrollment is now completed in both of our Phase 3 studies, CHERISH (childhood-onset) and ENDEAR (infant-onset), as well as our Phase 2 EMBRACE study. These are important milestones for the nusinersen program and both Phase 3 studies remain on track to complete in the first half of 2017.

The open-label SHINE study remains open for patients who have completed participation in ENDEAR and CHERISH, as well as patients who have completed the open-label Phase 2 study in childhood-onset SMA patients.

Ionis recently presented additional data from the open-label Phase 2 study in infants at the American Academy of Neurology Meeting. While we continue to be optimistic about these data, our well-controlled Phase 3 studies (CHERISH and ENDEAR) are designed to provide the data necessary for regulatory review and approval.

Additionally, the primary and secondary endpoints in our ENDEAR clinical trial protocol were recently updated. The decision to change the endpoints was carefully made based on additional insight from our open-label studies and discussions with regulators and experts in the field over the last year. The ENDEAR study was initially designed with a primary endpoint of permanent ventilation-free survival, meaning we would observe patients treated with 13 months of nusinersen compared to untreated patients and evaluate the number of patients who survive without the need for permanent ventilation between the two groups. It became increasingly clear that measuring motor milestones, endpoints of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders ("CHOP INTEND") and Compound Muscle Action Potential (CMAP), could be useful indicators of nusinersen’s potential efficacy in the ENDEAR study. Therefore, study’s primary endpoint has now been changed to add a second primary endpoint: motor milestones.

Full text: http://www.curesma.org/news/biogen-ionis-spring-2016-update.html

International map of the SMA

You can register myself on the international map of the SMA. Or search with it help of family with SMA for communication and exchange of experience.

I'm already here and I am SMA consultant https://www.diseasemaps.org/en/spinal-muscular-atrophy/