Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation
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Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation

Seelos Therapeutics has recently received the Rare Pediatric Disease Designation for their Sanfilippo syndrome treatment, trehalose. This designation is an incentive for companies to make therapies for conditions with an…

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Company Raises $14 Million Towards Sanfilippo Syndrome and Other Rare Disease Research
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Company Raises $14 Million Towards Sanfilippo Syndrome and Other Rare Disease Research

According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…

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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant
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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant

  According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…

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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy
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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy

  According to BioSpace, healthcare technology company RDMD has raised $14 million in Series A financing, and developed a partnership with UCB. In addition to growing their platform to discuss…

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Interim Gene Therapy Trial Data for Two Different Forms of Sanfilippo Syndrome Holds Promise
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Interim Gene Therapy Trial Data for Two Different Forms of Sanfilippo Syndrome Holds Promise

According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…

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Family is Determined to Make Lasting Memories with Daughter Diagnosed with Sanfilippo Syndrome
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Family is Determined to Make Lasting Memories with Daughter Diagnosed with Sanfilippo Syndrome

A family is grieving for their daughter and taking it one day at a time in the face of a terminal diagnosis.   Kelly and Andrew Mills became pregnant with…

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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?

According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…

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Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome
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Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome

Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…

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Can a Brain Implant Treat Dementia Caused by Sanfilippo Syndrome? A Boy’s Story Offers Hope
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Can a Brain Implant Treat Dementia Caused by Sanfilippo Syndrome? A Boy’s Story Offers Hope

According to a story from metro.co.uk, Harley Bond was three years old when he was first diagnosed with Sanfilippo syndrome type B, a rare progressive genetic disorder. Now five years…

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IND Accepted for an Experimental Sanfilippo Syndrome Treatment
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IND Accepted for an Experimental Sanfilippo Syndrome Treatment

According to a story from forextv.com, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND)…

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Tickets are on Sale Now for Rare Disease Film Festival in Boston

Are we ready for our close-up? The second annual Selections from DISORDER: The Rare Disease Film Festival at Biotech Week Boston will be held at the Takeda Auditorium in Cambridge,…

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Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress
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Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress

According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…

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Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress
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Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress

According to a story from globenewswire.com, the biopharmaceutical company Abeona Therapeutics, Inc. recently issued an announcement in regards to the company's Phase 1/2 clinical trial, which is testing the company's…

Continue Reading Sanfilippo Syndrome Type B (MPS IIIB) Clinical Trial Continues to Progress
Experimental Treatment for Sanfilippo Syndrome Type B Gets Fast Track Designation
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Experimental Treatment for Sanfilippo Syndrome Type B Gets Fast Track Designation

According to a story from finanznachrichten.de, the biopharmaceutical company Abeona Therapeutics, Inc. recently announced that its experimental medical product ABO-101 has earned Fast Track designation from the US Food and…

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Bioscience Companies in Ohio Take Aim at Rare Diseases
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Bioscience Companies in Ohio Take Aim at Rare Diseases

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

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New Collaborative Effort Will Test a Potential Treatment for Sanfilippo Syndrome
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New Collaborative Effort Will Test a Potential Treatment for Sanfilippo Syndrome

According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…

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Licensing Agreement Struck for an Experimental Sanfilippo Syndrome Drug
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Licensing Agreement Struck for an Experimental Sanfilippo Syndrome Drug

According to a story from publicnow.com, the drug development company Sarepta Therapeutics recently announced that is has completed negotiations for a licensing agreement with Lysogene, a biopharmaceutical company. This agreement…

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After Reviewing Publications About MPS III, Researchers Say More Studies Need to be Carried Out
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After Reviewing Publications About MPS III, Researchers Say More Studies Need to be Carried Out

Researchers have carried out a literature review to investigate MPS III. After reviewing forty-six papers, they concluded that more research needs to be carried out. You can find the original study…

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An Algorithm That Uses Facial Features to Diagnose Sanfilippo Syndrome Type-B Has Been Developed
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An Algorithm That Uses Facial Features to Diagnose Sanfilippo Syndrome Type-B Has Been Developed

Collaboration between the FDNA, the Cure Sanfilippo Foundation, and the Jonah’s Just Begun Foundation has led to technology that successfully recognises the facial phenotype (observable characteristics) of patients with mucopolysaccharidosis…

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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.
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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.

A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…

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The Merrill Family Faces the Challenges of Sanfilippo Syndrome Every Day. Now They Are Fundraising to Help Others
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The Merrill Family Faces the Challenges of Sanfilippo Syndrome Every Day. Now They Are Fundraising to Help Others

According to a story from tapinto.net, the Merrill family was crushed upon learning that their five-year-old daughter Leila was born with Sanfilippo syndrome, a lysosomal storage disease that ultimately causes…

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