I wish everyone with Happy Merry Christmas!
My name is Monika Lemeshonok, I am painter from Belarus. From born I have rare disease of the spinal cord - Spinal Muscular Atrophy Type 2. From this disease, I can’t walk and move, I'm in a wheelchair. But I love life and around the world. I express my love for life through painting artwork, I have art exhibitions throughout Europe and many art lovers appreciate my works.
Monday, December 24, 2018
Thursday, November 1, 2018
Started SMA charity marathon
I congratulate everyone on Happy Halloween! Yesterday you helped me and you bought 2 of my pictures. Thank you for that!
But our SMA charity marathon continues.
Buy 10 my pictures every day for a month and you will help me for my treatment. Click and buy right now:https://promote.shutterstock.com/widget/6c0d04d5-5e6d-4622-9f54-de29d886538f/share?fbclid=IwAR0yNpkCGkMl7P-R5Bo-9KRgV-dGXHpg62y_fy4xS1u_cj0C7fV1OuhrjBU
But our SMA charity marathon continues.
Buy 10 my pictures every day for a month and you will help me for my treatment. Click and buy right now:https://promote.shutterstock.com/widget/6c0d04d5-5e6d-4622-9f54-de29d886538f/share?fbclid=IwAR0yNpkCGkMl7P-R5Bo-9KRgV-dGXHpg62y_fy4xS1u_cj0C7fV1OuhrjBU
Sunday, October 28, 2018
Very GOOD NEWS about gene therapy Avexis
Patient enrollment is complete for AveXis Phase 1 clinical trial testing its gene therapy candidate AVXS-101 in patients with spinal muscular atrophy (SMA) type 2.
“We are pleased that STRONG is now fully-enrolled and expect to report data from this study by May 2019,” David Lennon, PhD, the company’s president, said in an email reply to SMA News Today.
The trial, named STRONG (NCT03381729), will test the safety and tolerability of single-dose intrathecal injection (into the spinal canal) of 27 patients up to 60 months (5 years) of age who are able to sit but not stand or walk.
Evaluation of the higher dose (1.2 X 10^14 vg) will depend on the safety of the lower dose (6.0 X 10^13 vg), each initially tested in three patients. Patients will be divided into two groups: those younger or older than two years at time of dosing. Effectiveness will be evaluated after 12 months of follow-up.
AVXS-101 is designed to deliver a functional copy of the SMN1 gene to cells that control muscle contractions, called motor neurons. SMN1 is defective in SMA patients, leading to lower levels of a working SMN protein, and subsequently to loss of motor neurons, progressive muscle weakness, and atrophy (shrinkage).
All patients in the STRONG trial have SMN1 gene’s exon 7 deleted in both copies, or alleles. Exons are the bits of DNA with information to generate proteins. These patients also have three copies of the SMN2 gene, which produces an unstable and shorter version of the SMN protein.
AVXS-101 is now under review for approval inthe U.S. and Europe for intravenous (IV) delivery in infants up to 9 months of age who have SMA type 1. A pre-application review period also was started in Japan. Final decisions on whether to approve AVXS-101 are expected in mid-2019.
Recently acquired by Novartis, AveXis has been working to ensure adequate supply of AVXS-101 to patients in case it is approved by the U.S. Food and Drug Administration (FDA), Lennon said.
The recent filings were based mainly on the open-label, dose-escalation Phase 1 START trial (NCT02122952), testing the safety, tolerability and effectiveness of two IV-delivered doses of AVXS-101 in 15 babies with SMA type 1, the most severe and common type of the disease.
“Compared to natural history, AVXS-101 delivered rapid improvement in motor milestone achievements, a dramatic survival benefit, and a durable effect going out four years in SMA type 1 patients,” Lennon said.
Early results of the ongoing Phase 3 STR1VE trial (NCT03306277) in type 1 children younger than 6 months showed improvements in movement ability, as well as no need for respiratory or nutritional support.
Although not part of the current applications for approval, older children with SMA type 2, such as those in STRONG, as well as those with SMA type 3, may be the subject of future filings. “Based on clinical results to date for AVXS-101 in SMA type 1, we expect AVXS-101 delivered via IT [intrathecal] administration to also show a positive benefit in [other] SMA subtypes,” Lennon said.
Besides providing information on the potential benefits of AVXS-101 in SMA type 2, STRONG also will help define the next steps of AveXis’ program. “Because it is our first study of the IT formulation of AVXS-101, data from STRONG will also help determine the final design for our planned study in children up to 18 years of age with SMA types 1, 2 and 3,” Lennon said.
This trial, called REACH, is set to begin in 2019 and will include patients ineligible for the company’s other studies.
Overall, this illustrates the company’s aim to expand the indications treated by AVXS-101. In a February interview with SMA News Today, Suku Nagendran, then AveXis chief medical officer, mentioned how Spinraza (nusinersen, by Biogen) progressed over time as a suggestion of what may lie ahead for AVXS-101.
“We are pleased that STRONG is now fully-enrolled and expect to report data from this study by May 2019,” David Lennon, PhD, the company’s president, said in an email reply to SMA News Today.
The trial, named STRONG (NCT03381729), will test the safety and tolerability of single-dose intrathecal injection (into the spinal canal) of 27 patients up to 60 months (5 years) of age who are able to sit but not stand or walk.
Evaluation of the higher dose (1.2 X 10^14 vg) will depend on the safety of the lower dose (6.0 X 10^13 vg), each initially tested in three patients. Patients will be divided into two groups: those younger or older than two years at time of dosing. Effectiveness will be evaluated after 12 months of follow-up.
AVXS-101 is designed to deliver a functional copy of the SMN1 gene to cells that control muscle contractions, called motor neurons. SMN1 is defective in SMA patients, leading to lower levels of a working SMN protein, and subsequently to loss of motor neurons, progressive muscle weakness, and atrophy (shrinkage).
All patients in the STRONG trial have SMN1 gene’s exon 7 deleted in both copies, or alleles. Exons are the bits of DNA with information to generate proteins. These patients also have three copies of the SMN2 gene, which produces an unstable and shorter version of the SMN protein.
AVXS-101 is now under review for approval inthe U.S. and Europe for intravenous (IV) delivery in infants up to 9 months of age who have SMA type 1. A pre-application review period also was started in Japan. Final decisions on whether to approve AVXS-101 are expected in mid-2019.
Recently acquired by Novartis, AveXis has been working to ensure adequate supply of AVXS-101 to patients in case it is approved by the U.S. Food and Drug Administration (FDA), Lennon said.
The recent filings were based mainly on the open-label, dose-escalation Phase 1 START trial (NCT02122952), testing the safety, tolerability and effectiveness of two IV-delivered doses of AVXS-101 in 15 babies with SMA type 1, the most severe and common type of the disease.
“Compared to natural history, AVXS-101 delivered rapid improvement in motor milestone achievements, a dramatic survival benefit, and a durable effect going out four years in SMA type 1 patients,” Lennon said.
Early results of the ongoing Phase 3 STR1VE trial (NCT03306277) in type 1 children younger than 6 months showed improvements in movement ability, as well as no need for respiratory or nutritional support.
Although not part of the current applications for approval, older children with SMA type 2, such as those in STRONG, as well as those with SMA type 3, may be the subject of future filings. “Based on clinical results to date for AVXS-101 in SMA type 1, we expect AVXS-101 delivered via IT [intrathecal] administration to also show a positive benefit in [other] SMA subtypes,” Lennon said.
Besides providing information on the potential benefits of AVXS-101 in SMA type 2, STRONG also will help define the next steps of AveXis’ program. “Because it is our first study of the IT formulation of AVXS-101, data from STRONG will also help determine the final design for our planned study in children up to 18 years of age with SMA types 1, 2 and 3,” Lennon said.
This trial, called REACH, is set to begin in 2019 and will include patients ineligible for the company’s other studies.
Overall, this illustrates the company’s aim to expand the indications treated by AVXS-101. In a February interview with SMA News Today, Suku Nagendran, then AveXis chief medical officer, mentioned how Spinraza (nusinersen, by Biogen) progressed over time as a suggestion of what may lie ahead for AVXS-101.
Wednesday, September 19, 2018
I need your help!
Dear, friends!
I must tell you the sad news! 😥
Unfortunately I could not collect the necessary amount for a charity account for my trip to the clinic in Bologna. Therefore, my trip is canceled this year. I'm very upset and frustrated from this event because my health is deteriorating and it's difficult for me to breathe periodically. But I have to reschedule my trip for May 2019. I say "Thank you very much!" to those who have already sent part of the charitable funds for my treatment!
NOW I again continue collecting charitable donations for my treatment. This amount includes treatment and procedures at the Nigrisoli clinic, the cost of air tickets (for 3 people, I, my mother and grandmother), the cost of my orthopedic corset and orthopedic pillows-holders for the back for my stroller. The total cost of all this is 2000 euros.
I ask you to help me all of you to make a charitable donation or make a repost!
My charity account: PayPal: Lemeshonok@gmail.com
I must tell you the sad news! 😥
Unfortunately I could not collect the necessary amount for a charity account for my trip to the clinic in Bologna. Therefore, my trip is canceled this year. I'm very upset and frustrated from this event because my health is deteriorating and it's difficult for me to breathe periodically. But I have to reschedule my trip for May 2019. I say "Thank you very much!" to those who have already sent part of the charitable funds for my treatment!
NOW I again continue collecting charitable donations for my treatment. This amount includes treatment and procedures at the Nigrisoli clinic, the cost of air tickets (for 3 people, I, my mother and grandmother), the cost of my orthopedic corset and orthopedic pillows-holders for the back for my stroller. The total cost of all this is 2000 euros.
I ask you to help me all of you to make a charitable donation or make a repost!
My charity account: PayPal: Lemeshonok@gmail.com
Monday, August 13, 2018
Memory Days of SMA
Today is the Memory Days of remembe of people who passed away from this world from the terrible diagnosis SMA. I remember all of you, my friends.
Thursday, May 31, 2018
The shock news! Roche ends development of olesoxime
It was announced today that Roche has taken the difficult decision of ending the development of neuro-protective compound Olesoxime.
In 2016 Roche started the OLEOS study, an open label extension study evaluating the long term safety and effectiveness of olesoxime. Many discussions with Health Authorities (FDA and EMA) and SMA experts were had and the company worked hard to improve the dose and formulation of olesoxime and to design the best Phase 3 study possible to be able to show the efficacy of olesoxime.
Furthermore, they regularly analysed the data from this study. Whilst the data at 12 months of treatment with olesoxime were initially encouraging, the most recent analysis at 18 months, which was presented at the American Academy of Neurology in April 2018, actually showed a worsening in motor function.
Unfortunately, despite all of their efforts and a strong desire to deliver olesoxime as a medicine to people with SMA, Roche has concluded that this is not going to be possible. Based on all of the available evidence and the continued difficulties described above, the company has decided to stop further development of olesoxime.
Many of you will be very disappointed by this news, as we are too. Their immediate priority now is to ensure that those still taking part in the ongoing OLEOS study understand what this decision means for them and that they are provided with appropriate treatment options. Roche is working closely with study sites and investigators to help identify options for those still taking part in the OLEOS study and will share more details about this in the coming weeks. The OLEOS study will be kept open until all ongoing participants have an alternative treatment option confirmed.
text from: http://www.sma-europe.eu/news/roche-ends-development-of-olesoxime/
In 2016 Roche started the OLEOS study, an open label extension study evaluating the long term safety and effectiveness of olesoxime. Many discussions with Health Authorities (FDA and EMA) and SMA experts were had and the company worked hard to improve the dose and formulation of olesoxime and to design the best Phase 3 study possible to be able to show the efficacy of olesoxime.
Furthermore, they regularly analysed the data from this study. Whilst the data at 12 months of treatment with olesoxime were initially encouraging, the most recent analysis at 18 months, which was presented at the American Academy of Neurology in April 2018, actually showed a worsening in motor function.
Unfortunately, despite all of their efforts and a strong desire to deliver olesoxime as a medicine to people with SMA, Roche has concluded that this is not going to be possible. Based on all of the available evidence and the continued difficulties described above, the company has decided to stop further development of olesoxime.
Many of you will be very disappointed by this news, as we are too. Their immediate priority now is to ensure that those still taking part in the ongoing OLEOS study understand what this decision means for them and that they are provided with appropriate treatment options. Roche is working closely with study sites and investigators to help identify options for those still taking part in the OLEOS study and will share more details about this in the coming weeks. The OLEOS study will be kept open until all ongoing participants have an alternative treatment option confirmed.
text from: http://www.sma-europe.eu/news/roche-ends-development-of-olesoxime/
Wednesday, May 16, 2018
AveXis provides community update on plans for AVXS-101 in Europe
AveXis has provided SMA Europe with a community update on plans for AVXS-101 in Europe. AVXS-101 is a gene therapy product for SMA.
STR1VE EU
OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centres across the European Union.
ADMINISTRATION: In STR1VE EU, AVXS-101 is administered through a one-time IV infusion.
WHO: STR1VE EU will enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy.
SPRINT
OVERVIEW: SPRINT is expected to start enrolling patients in the first half of 2018 in presymptomatic patients with SMA Types 1, 2 and 3.
ADMINISTRATION: In SPRINT, AVXS-101 is administered through a one-time IV infusion.
WHO: SPRINT is expected to enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy.
REACH
OVERVIEW: REACH is expected to start enrolling patients late in 2018 or early in 2019 in
patients with SMA Types 1, 2 and 3.
ADMINISTRATION: In REACH, AVXS-101 is administered through a one-time IT injection. Data from STRONG (the first study of AVXS-101 delivered through IT injection) will help determine the final study design.
WHO: REACH is expected to enroll approximately 50 patients with SMA Types 1, 2 and 3 who are between approximately six months and 18 years of age.
Text from: http://www.sma-europe.eu/news/avexis-provides-community-update-on-plans-for-avxs-101-in-europe/
STR1VE EU
OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centres across the European Union.
ADMINISTRATION: In STR1VE EU, AVXS-101 is administered through a one-time IV infusion.
WHO: STR1VE EU will enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy.
SPRINT
OVERVIEW: SPRINT is expected to start enrolling patients in the first half of 2018 in presymptomatic patients with SMA Types 1, 2 and 3.
ADMINISTRATION: In SPRINT, AVXS-101 is administered through a one-time IV infusion.
WHO: SPRINT is expected to enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of gene therapy.
REACH
OVERVIEW: REACH is expected to start enrolling patients late in 2018 or early in 2019 in
patients with SMA Types 1, 2 and 3.
ADMINISTRATION: In REACH, AVXS-101 is administered through a one-time IT injection. Data from STRONG (the first study of AVXS-101 delivered through IT injection) will help determine the final study design.
WHO: REACH is expected to enroll approximately 50 patients with SMA Types 1, 2 and 3 who are between approximately six months and 18 years of age.
Text from: http://www.sma-europe.eu/news/avexis-provides-community-update-on-plans-for-avxs-101-in-europe/
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