Amit Rakhit

@AmitR_nyc

biotech Chief Medical Officer, Pedi Cardiologist, champion for people with rare diseases, human rights supporter, cofounder KoMocean, foodie, traveler

New York, NY
Vrijeme pridruživanja: veljača 2009.

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  1. proslijedio/la je Tweet
    23. pro 2019.

    would like to wish the community a !

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  2. proslijedio/la je Tweet
    24. pro 2019.

    A message from to the patients, families, and community that we serve. On behalf of every employee at , we wish you a joyful holiday season and a very Happy New Year.

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  3. proslijedio/la je Tweet
    19. pro 2019.

    An diagnosis can be extremely impactful on a family, changing their future. Hear how one family is embracing each day with hope, thanks to help from their friends and local community.

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  4. proslijedio/la je Tweet
    20. pro 2019.

    We were glad to sponsor the Patient-Focused Drug Development meeting on developmental & epileptic encephalopathies including hosted by last month. To hear insights from caregivers or to watch the moving opening video, visit .

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  5. proslijedio/la je Tweet
    21. pro 2019.

    For people living with , , and are very common. Extreme emotions and over-excitement can be a struggle to manage. is developing new medicines for families living with Angelman syndrome. We’re listening.

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  6. proslijedio/la je Tweet
    11. pro 2019.

    We support the efforts of 25+ national & international organizations in recognizing Infantile Spasms Awareness Week. Please retweet to help raise awareness among families as well as medical professionals. Learn more about at .

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  7. proslijedio/la je Tweet
    12. pro 2019.

    Thank you to & The Loulou Foundation for organizing a meeting to enable families to share their perspective on the impact of Deficiency on daily life with researchers & officials at . Watch the video at .

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  8. proslijedio/la je Tweet
    13. pro 2019.

    In a video by , , VP of Patient Engagement & Government Affairs, shares the importance of infusing what matters most to families in every element of the discovery & development of new treatments for .

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  9. proslijedio/la je Tweet
    9. pro 2019.

    That wraps up our ! If you’d like to ask a question, reply below and we’ll try to follow-up. ()

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  10. proslijedio/la je Tweet
    9. pro 2019.

    We’ve gotten a few questions regarding locations of our studies, especially regarding NEPTUNE and specifically regarding international locations. At this time, sites in the US are San Diego (CA), Tacoma (WA), Media (PA) and Phoenix (AZ). ()

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  11. proslijedio/la je Tweet
    9. pro 2019.

    In that study, we saw an improvement in overall global functioning as measured by a specific scale called the CGI-I. This helped inform the design of our Phase 3 NEPTUNE study, after discussions with the FDA. ()

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  12. proslijedio/la je Tweet
    9. pro 2019.

    Now, what does that mean? OV101 had no significant safety and tolerability concerns and had improvements in several areas of an individual’s functioning. ()

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  13. proslijedio/la je Tweet
    9. pro 2019.

    The Study met safety and tolerability endpoints as well as achieving a statistically significant (p= 0.0006) efficacy endpoint in the study. ()

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  14. proslijedio/la je Tweet
    9. pro 2019.

    Let’s talk about STARS, the first industry-sponsored, international, randomized, double-blind, placebo-controlled in adults and adolescents with . ()

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  15. proslijedio/la je Tweet
    9. pro 2019.

    We’re excited that enrollment is open for all ages (4 – 12 years old) and we look forward to the results from the Phase 3 NEPTUNE in mid-2020. ()

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  16. proslijedio/la je Tweet
    9. pro 2019.

    Additionally, the independent data monitoring committee’s review of the safety and dosing information in the Phase 3 NEPTUNE study corroborates our dose modeling that guided the dosing paradigm in the . ()

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  17. proslijedio/la je Tweet
    9. pro 2019.

    After a predetermined review by the independent data monitoring committee (IDMC) of the NEPTUNE safety and PK information. The IDMC recommended opening enrollment to the younger age cohort (ages 4-8). ()

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  18. proslijedio/la je Tweet
    9. pro 2019.

    At , I announced progress in the ongoing clinical development of OV101, our investigational therapy for and syndrome. ()

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  19. proslijedio/la je Tweet
    9. pro 2019.

    Hello everyone. Happy to take over Ovid’s Twitter handle for a quick to discuss further some of the information I presented at last Friday. ()

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  20. proslijedio/la je Tweet
    9. pro 2019.

    Let me take this opportunity to thank for having us. Our team was able to meet with quite a few patients and caregivers. We always look forward to opportunities to speak with the community. ()

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