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Daily Archives: June 24, 2021

Home » Archives for June 24, 2021
Remarkable Results From a Phase 2 Trial for Beta Thalassemia
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Remarkable Results From a Phase 2 Trial for Beta Thalassemia

  • Post author:Sunniva Bean
  • Post published:June 24, 2021
  • Post category:Beta Thalassemia

As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…

Continue Reading Remarkable Results From a Phase 2 Trial for Beta Thalassemia
Filsuvez for EB Granted Priority Review 
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Filsuvez for EB Granted Priority Review 

  • Post author:Jessica Lynn
  • Post published:June 24, 2021
  • Post category:Epidermolysis Bullosa

In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…

Continue Reading Filsuvez for EB Granted Priority Review 
RDLA Webinar: The HEART Act, Newborn Screening, and More
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RDLA Webinar: The HEART Act, Newborn Screening, and More

  • Post author:James Moore
  • Post published:June 24, 2021
  • Post category:Rare Disease

The Rare Disease Legislative Advocates (RDLA) webinar for the month of June took place on June 17, 2021. This month's webinar provided updates on a variety of legislative topics that…

Continue Reading RDLA Webinar: The HEART Act, Newborn Screening, and More
Compassion Corner: Compassion, Empathy, Sympathy, and Control in a Clinical Setting
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Compassion Corner: Compassion, Empathy, Sympathy, and Control in a Clinical Setting

  • Post author:Rose Duesterwald
  • Post published:June 24, 2021
  • Post category:Rare Disease

Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…

Continue Reading Compassion Corner: Compassion, Empathy, Sympathy, and Control in a Clinical Setting
FDA Approves Revolo’s ‘1104 IND for EoE
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FDA Approves Revolo’s ‘1104 IND for EoE

  • Post author:Jessica Lynn
  • Post published:June 24, 2021
  • Post category:Eosinophilic Esophagitis

In a news release from June 23, 2021, Revolo Biotherapeutics ("Revolo") shared that the FDA approved an Investigational New Drug (IND) application for the company's '1104 peptide treatment. Altogether, '1104…

Continue Reading FDA Approves Revolo’s ‘1104 IND for EoE
This Grandfather Bikes to Raise Money for Held in Our Hearts
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This Grandfather Bikes to Raise Money for Held in Our Hearts

  • Post author:Kendall Mason
  • Post published:June 24, 2021
  • Post category:Congenital Nephrotic Syndrome

Mark Dalziel has big plans for this weekend; he's going to bike the length of Scotland within two days. He has a very good reason for doing so, which is…

Continue Reading This Grandfather Bikes to Raise Money for Held in Our Hearts
PT001 for PAH Earns Orphan Drug Status
InspiredImages / Pixabay

PT001 for PAH Earns Orphan Drug Status

  • Post author:Jessica Lynn
  • Post published:June 24, 2021
  • Post category:pulmonary arterial hypertension

  Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News,…

Continue Reading PT001 for PAH Earns Orphan Drug Status
Raising MdDS Awareness: Brandy’s Story
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Raising MdDS Awareness: Brandy’s Story

  • Post author:Jessica Lynn
  • Post published:June 24, 2021
  • Post category:Mal de debarquement

  Mal de débarquement, or "sickness of disembarkment," is a phenomenon which causes a bobbing, rocking, or swaying sensation, alongside other symptoms. With many people, mal de débarquement only lasts…

Continue Reading Raising MdDS Awareness: Brandy’s Story
The Rare 2030 Action Campaign For A Better Rare Future

The Rare 2030 Action Campaign For A Better Rare Future

  • Post author:Sunniva Bean
  • Post published:June 24, 2021
  • Post category:Rare Disease

As reported in Charcot-Marie Tooth News; this year, Eurordis has kicked off a campaign to make sure those with rare diseases don’t get left behind in the coming century. Small…

Continue Reading The Rare 2030 Action Campaign For A Better Rare Future

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