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November 19, 2020 News by Marisa Wexler, MS

Taysha, Invitae Partner to Offer Genetic Testing for Lysosomal Storage Disorders, Epilepsy

Taysha Gene Therapies and Invitae have announced a partnership to support Invitae’s Detect Lysosomal Storage Diseases (Detect LSDs) and Behind the Seizure programs by offering free genetic testing to patients. The Detect LSDs program aims to enable the rapid diagnosis of lysosomal storage diseases such as…

March 22, 2019 News by Alice Melão

Team Sanfilippo Planning to Launch Phase 2 Trial Testing Trehalose as Sanfilippo Treatment

Nonprofit research foundation Team Sanfilippo (TSF) is preparing to launch a Phase 2 clinical trial to explore the safety and efficacy of a common sugar called trehalose as a treatment for patients with Sanfilippo syndrome. This follows a collaborative agreement that was initially made with Bioblast Pharma, the therapy’s…

Recent Posts

  • Seeing death and suffering makes me fear for my child’s future
  • Sanfilippo B therapy JR-446 gets orphan drug status in Europe
  • I hadn’t anticipated being a stay-at-home mom, but it works for us
  • Sanfilippo B enzyme replacement therapy wins orphan drug status
  • Trying to meet my son’s needs so he doesn’t feel like a ‘glass child’
  • Balancing logic and emotion isn’t easy as a Sanfilippo parent
  • Spruce to move tralesinidase alfa into Phase 3 clinical study
  • I’m hopeful about the future of Sanfilippo syndrome type B
  • HSPC transplant eases disease symptoms in Sanfilippo C mice
  • Forever a toddler: Grieving the independent child I’d dreamed of


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