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

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.

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/

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/

FDA Grants Orphan Drug Status to SRK-015 for SMA

SRK-015 has received orphan drug status designation by the U.S. Food and Drug Administration (FDA) to treat muscle atrophy in patients with spinal muscular atrophy (SMA).

SRK-105 is Scholar Rock’s lead product candidate, intended to improve muscle strength and motor function in SMA patients. The potential therapy selectively inhibits the activation of myostatin, a protein mainly produced in skeletal muscle cells that suppresses muscle growth.

Absence of the MSTN gene, which codes for myostatin, is associated with increased muscle mass and strength in animal models. Based on preclinical evidence from animal models, scientists at Scholar Rock believe that inhibiting the activation of myostatin will promote a similar, clinically meaningful effect.

Mice studies showed that SRK-105 can prevent additional atrophy in animals with muscle wasting and can improve muscle mass and function. Promising findings were also reported in studies with primates, where the investigational compound was able to increase animals’ lean body mass, particularly in a type of muscle fiber affected by SMA.

These preclinical studies support the company’s decision to advance into clinical testing in SMA patients.

According to Scholar Rock, SRK-105 may become the first muscle-targeting treatment to reverse or prevent muscle atrophy in patients with SMA. It could be used both as a stand-alone therapy and as a combination treatment with the current standard of care.

“We are very pleased that the FDA granted Orphan Drug Designation to SRK-015 for the treatment of patients suffering from SMA, and we appreciate that the agency’s decision came much earlier than anticipated,” Nagesh Mahanthappa, PhD, President and CEO of Scholar Rock, said in a press release.
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“This designation is an important milestone in the development of our lead product candidate along the path towards a first-in-human Phase 1 clinical trial in the second quarter of 2018,” Mahanthappa added.

Orphan drugs are intended for the treatment, diagnosis or prevention of rare diseases affecting fewer than 200,000 people in the United States. The designation also can be granted for diseases affecting more than 200,000 people if there is no reasonable expectation that the company will be able to recover the costs of drug development and marketing.

The designation is granted if there is a medically plausible basis for using the treatment candidate. Orphan drug status provides incentives for product development, including tax credits for clinical trials, exemption from a prescription drug user fee, and access to protocol assistance from the FDA.

Companies may also benefit from seven years of market exclusivity if the drug is ultimately approved.

Text from:https://smanewstoday.com/2018/04/06/promising-sma-therapy-srk-015-gains-orphan-drug-status-from-fda/?amp

My Easter charity event

Dear, friends!

Long time I don't wrote here, because I had problem with my spine, again I had spinal pain, its very terrible, but now me better and I am here.

Now I want to say that in May I will have to go to a clinic in Bologna for treatment, my doctor told me about it. This treatment will improve my condition. But now I need your help! I need charitable funds to buy plane tickets to Italy for treatment.

In soon time will be Easter and I am now announcing charity event. In this online gallery sale of my artworks. If you want to help me you can buy my paintings here: https://www.saatchiart.com/account/artworks/60435

I will be very grateful if you will help me find funds for treatment and travel.

AveXis Plans a May Start for Phase 3 European Trial of Its SMA Type 1 Gene Therapy

AveXis’ expects to start a Phase 3 clinical trial of its spinal muscular atrophy (SMA) gene therapy AVXS-101 in Europe in May.

The STR1VE-EU trial (NCT03461289) is expected to include up to 30 children under 6 months of age with type 1 SMA, the disease‘s most severe and common form. All will receive a single dose of intravenous AVXS-101.

The therapy’s safety and effectiveness will be assessed regularly until the babies reach 18 months. At that point, their parents can allow those who are eligible to take part in a long-term follow-up study.

Researchers will be looking at whether the infants can sit without support and also the number who survive until 14 months of age.

The trial will be conducted in Belgium, France, Germany, Italy, the Netherlands, Spain, Sweden, and the U.K. It is expected to be completed in November 2020.

AVXS-101 contains a normal version of the SMN gene that is defective in SMA. Movement nerve cells need a normal version to survive and thrive.

AveXis delivers the gene with a non-infectious virus. The company designed it to be a one-time treatment to prevent muscle degeneration over a lifetime, it said in a recent interview with SMA News Today.

Babies who take part in the STR1VE-EU trial must have mutations of both SMN1 alleles and one or two copies of SMN2. Alleles are gene variations. In most cases, the more copies a patient has of SMN2, the less severe the disease will be.

AveXis has already conducted a Phase 1 trial (NCT02122952) of AVXS-101 in 15 type 1 SMA infants, who received the therapy before they reached six months of age.

The key finding was that all of the children survived for 20 months. This was a huge improvement over what normally happens — 92 percent of children dying by that age.

Another stunning result was that 11 of the 12 children who received the high dose of the therapy were able to sit unassisted, eat food through their mouth, and speak. Nine were able to roll over, and two could walk without help.

AveXis continues to recruit participants for its ongoing STR1VE Phase 3 trial (NCT03306277) in the United States. The trial is also looking at AVXS-101’s safety and effectiveness in children with type 1 SMA who are less than 6 months old. Results similar to those seen in the completed Phase 1 study were observed in the first three children dosed, AveXis said.

Meanwhile, the company continues to recruit patients for its ongoing STRONG Phase 1 trial (NCT03381729) in children up to 60 months of age with type 2 SMA. Unlike the studies in type 1 SMA, which use intravenous therapy administration, patients in the STRONG trials will receive AVXS-101 in the spinal canal, which enables a more targeted treatment.

AveXis also plans the SPRINT trial program for infants under 6 weeks of age with no symptoms but who are likely to develop SMA types 1, 2, or 3, and the REACH program for types 1-3 SMA patients ages 6 months to 18 years who are ineligible for the other studies.

SPRINT and REACH will be worldwide trials. SPRINT is expected to start by mid-2018 and REACH by late 2018 or early 2019.

AveXis and France-based Genethon, which developed AVXS-101, recently announced an agreement granting AveXis the rights to patents in the U.S., Europe and Japan that cover Genethon’s AAV9 SMN gene therapy technology and delivery system. They cover delivery by IV and the spinal canal.

Text from:https://smanewstoday.com/2018/03/23/avexis-sma-gene-therapy-european-phase-3-trial-to-start-in-may/?utm_content=bufferf6d7e&utm_medium=organic+social&utm_source=facebook.com&utm_campaign=buffer