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What do you do when your child s life is dependent on a Rs 16 crore drug?

What do you do when your child’s life is dependent on a Rs 16 crore drug? © Provided by The Times of India Ayaansh was about eight months old when his family realised something was wrong he could not crawl or sit up, struggled to have milk or food, and had no neck control. The paediatrician would say that it could be a milestone delay. “But, when he did not achieve any milestone at nine months, we visited a neurologist and were shocked to hear that he had Spinal Muscular Atrophy (SMA),” says Yogesh Gupta, Ayaansh’s father. This was in June 2019. Two years on, the family’s hopes are pinned on a wonder drug - Zolgensma. However, the medicine costs $2.125m (around Rs 16 crore) are prohibitive. So far, the family has been able to raise Rs 1.4 crore and are requesting NGOs, philanthropists and the government to help.

Centogene N V (CNTG) Q4 2020 Earnings Call Transcript

Operator Good day, and thank you for standing by. Welcome to the CENTOGENE first-year 2020 earning results conference call. [Operator instructions] After the speaker s presentation, there would be a question-and-answer session. [Operator instructions] Please be advised that today s conference is being recorded. [Operator instructions] I would now like to hand the conference over to your speaker today, Lennart Streibel. Please go ahead. Lennart Streibel Investor Relations Thanks, Sarah. Hello and welcome. Thank you for joining us to discuss our Q4 and full-year 2020 results which were issued earlier today. You can view this presentation and the related press release on CENTOGENE s website.

REGENXBIO Announces Presentations at the American Society of Gene and Cell Therapy s 24th Annual Meeting

REGENXBIO Announces Presentations at the American Society of Gene and Cell Therapy s 24th Annual Meeting News provided by Share this article Share this article ROCKVILLE, Md., April 27, 2021 /PRNewswire/ REGENXBIO Inc. (Nasdaq: RGNX) today announced that four oral and nine poster presentations will be presented at the American Society of Gene and Cell Therapy s 24 th Annual Meeting taking place from May 11 to 14, 2021, in virtual format. Presentations and posters will be available at https://www.asgct.org/. Oral presentations include: Abstract Title: Effects of Sexual Dimorphism and Genetic Background on AAV Tissue Transduction in Mice Following Intravenous Administration of a Diverse Capsid Pool (abstract #94)

Takeda launches mobile application to support patient for treatment of genetic diseases

Takeda launches mobile application to support patient for treatment of genetic diseases By siliconindia  |  Friday, 26 February 2021, 12:16 Hrs Takeda India, part of the Takeda Pharmaceutical Company Limited, a global values-based, R&D-driven biopharmaceutical leader, launched the XPERT EASE application. The application is developed for the exclusive use of healthcare professionals to address awareness-related challenges and enable an efficient digital ecosystem for disease updates and dose calculation for improved management of patients with rare lysosomal storage disorders Doctors screening, diagnosis, and treatment of genetic diseases have often been identified as challenging, including advising the dosage of medicines specific to patients individual needs. The XPERT EASE is designed to keep in mind such challenges while managing lysosomal storage disorders.

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