Today international rare disease day

Hi, everyone!

Today international rare disease day.

I want say any words about me and my story. From 1.5 year old doctors says about my disease, about that I never will walk. But I not give up, because I always believed in miracle. All my life it's very strong fight, fight with SMA. It's fight go on every days. In my live I have meeting with different unique doctors.

Now I have many purposes, I want will doing surgery scoliosis, its very need for me. And also I dream that one day I go to on clinical trial gene therapy for patients with SMA. For me it is very important. But now I ask you don't remember about rare patient, we need your help.

I very need respiratory treatment in Bologna. I will be happy, if you send me your donation.

My charity account:
PayPal: Lemeshonok@gmail.com
VISA: 4246 4100 5470 2615

I am rare

Now rare week and this is post about it. I have rare disease Spinal Mucsular Atrophy and I would like that you support my сall. From SMA affected every 10000 child in the world.

Remember about people with rare disease, to us needed your help.

https://www.facebook.com/groups/1183688658388473/

"SMA Cure for Monika" has account in Instagram

Began rare week and now "SMA Cure for Monika" has account in Instagram.

I invitation everyone followings me: https://www.instagram.com/smacureformonika/

I need your support!

In soon time winter comes to an end, but I have not still charitable donation. Before my trip to Bologna is few months, but I still not have charitable funds for purchase tickets for my flight. I need to collect charitable 3000€ for my trip (buy tickets, visa, insurance, apartment rental).

I ask you to make repost this my message, and tell your friends about it urgently.

Last time I was very hard to sit back pain and scoliosis progresses. From this worsens my condition. 😭 When I'm going at the clinic in Bologna, I will ask to make a brace for my back. But I also do not have money for it. Only you can help me right now.

Charity account for donations:
PayPal: Lemeshonok@gmail.com
VISA: 4246 4100 5470 2615

My charitable group: https://www.facebook.com/groups/1183688658388473/

My invitation to Italiano-Belarusian Forum

Hi, friends!

Today I want to show my preview of the events that happened to me over the past few months after my trip to Bologna. In early December 2016, I was informed of the Italian Foundation "Aiutiamoli a Vivere" that neurologist Dr. M.Villanova from clinic Nigrisoli gave me a cough machine, to train my breathing. Me and my mother were invited to the Italian-Belarusian Forum which was held in Minsk, at the hotel "Planet". I was very pleased and surprised by this event. The evening of December 8 of us came home chairman of the Italian Fund "Aiutiamoli a Vivere" Signor Luciano Braconi and together we left in a car in Minsk, I was with my mother and grandmother.



On the morning of 9 December, we were invited to the grand opening of the Belarusian-Italian Forum. At the end of the forum my mother was invited to the stage and the Director of the Foundation Fabrizio Pacifici gave me a cough machine, he said it was a gift from Dr. Villanova. My mother told to audience about me and my future treatment in Italy. I was very happy this event.

For two months, every day I use breathing apparatus system cough and I feel improvement.

Watch our video about it.

Text from: https://www.facebook.com/groups/1183688658388473/

Again news from Avexis

CHICAGO, Feb. 06, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 in the European Union (EU) will reflect a single-arm design, using natural history of the disease as a comparator, and will enroll approximately 30 patients. This update is based on the receipt of the Scientific Advice response from the Scientific Advice Working Party within the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

In addition to evaluating safety the planned pivotal trial is expected to evaluate achievement of motor milestones, specifically patients’ ability to sit unassisted, as well as an efficacy measure defined by the time from birth to an “event,” defined as death or requiring at least 16 hours per day of ventilation support for breathing for greater than two weeks in the absence of an acute reversible illness, or perioperatively.

“The scientific advice process has been quite productive, with the EMA providing constructive guidance on a pivotal study design most appropriate for EU product registration,” said James L’Italien, Ph.D., Senior Vice President, Chief Regulatory and Quality Officer of AveXis. “We will utilize the enhanced communication pathways afforded to us via the PRIority MEdicines program and work in collaboration with the EMA to generate the high-quality data needed to support the most rapid submission that meets the requirements for marketing authorization in the EU.”

The CHMP additionally recommended AveXis discuss the potential for Conditional Marketing Authorization in a future meeting with EMA.

More specific clinical trial details will be made available at the time the study is initiated, which is expected in the second half of 2017.

The company expects to initiate a separate pivotal clinical trial of AVXS-101 in SMA Type 1 in the United States in the first half of 2017.

Text from:http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2243247

AveXis Gene Therapy AVXS-101 Granted Access into EMA PRIME Program for Spinal Muscular Atrophy Type 1!!

Great news from Avexis!

CHICAGO, Jan. 31, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the European Medicines Agency (EMA) has granted access into its PRIority MEdicines (PRIME) program for the company’s proprietary gene therapy, AVXS-101, for the treatment of spinal muscular atrophy (SMA) Type 1. The PRIME application was based on data from both preclinical evaluations and the ongoing Phase 1 clinical trial of AVXS-101 as of September 15, 2016.

PRIME is intended to enhance support for the development of medicines – specifically those that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options – through early and proactive support by EMA to optimize the generation of robust data and development plans, and potentially expedite the assessment of the Marketing Authorization Application (MAA) so these medicines may reach patients sooner.

“The acceptance of AVXS-101 into the PRIME program reflects the urgent need for innovative treatment options for the patients diagnosed with SMA in the European Union,” said James L’Italien, Ph.D., Senior Vice President, Chief Regulatory and Quality Officer of AveXis. “We are eager for this enhanced collaboration with the EMA to ensure we are taking the most appropriate and expeditious path toward the development of a robust Marketing Authorization Application submission, and to potentially streamlining the time needed to bring AVXS-101 to patients in the EU suffering from this devastating disease.”

In notifying the company of the acceptance, EMA noted: “The preliminary clinical observations following AVXS-101 administration include positive impact on survival, pulmonary function, nutritional support, preservation of motor function and the attainment of development milestones, all of which are unexpected within the framework of the natural history and disease progression for SMA Type 1. These clinically meaningful responses in the patients treated with AVXS-101 are sufficient preliminary clinical evidence of treatment effect that has the potential to address an unmet need in this devastating pediatric disease.”

In July 2016, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation, a comparable program to PRIME used by the FDA, for AVXS-101 for the treatment of patients with SMA Type 1.

AveXis intends to initiate a pivotal trial of AVXS-101 in patients with SMA Type 1 in the European Union before the end of 2017.

About PRIME

The EMA launched the PRIME initiative in March 2016 to foster research and development of medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to strengthen clinical trial designs to facilitate the generation of high quality data for the evaluation of an application for marketing authorization. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on preclinical and/or early clinical data. These medicines are considered priority medicines within the European Union.

After an investigational candidate has been selected for PRIME, developers are assigned a rapporteur from the Committee for Medicinal Products for Human Use (CHMP) to provide continuous support and help to build knowledge ahead of a Marketing Authorization Application (MAA). A multidisciplinary group of experts will provide broader guidance on the overall development plan and regulatory strategy of the product. Companies are also eligible for accelerated assessment at the time of their regulatory application.

For more information, please visit the research and development section of www.ema.europa.eu

Text from: http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2240880