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clinical trial

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Mepolizumab Improves Eosinophilic Asthma Symptoms, Study Shows
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Mepolizumab Improves Eosinophilic Asthma Symptoms, Study Shows

  • Post author:Jessica Lynn
  • Post published:June 28, 2021
  • Post category:Eosinophilic asthma

Eosinophilic asthma is a rare and often severe form of asthma which can be difficult to treat. Because of this, many patients with eosinophilic asthma experience diminished quality of life…

Continue Reading Mepolizumab Improves Eosinophilic Asthma Symptoms, Study Shows
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 
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
AT-007 for Galactosemia Granted Fast Track Designation
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AT-007 for Galactosemia Granted Fast Track Designation

  • Post author:Jessica Lynn
  • Post published:June 22, 2021
  • Post category:Galactosemia

According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…

Continue Reading AT-007 for Galactosemia Granted Fast Track Designation
New Research Shows How Pridopidine Works in HD
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New Research Shows How Pridopidine Works in HD

  • Post author:Jessica Lynn
  • Post published:June 17, 2021
  • Post category:Huntington's disease/Huntington's Disease

For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…

Continue Reading New Research Shows How Pridopidine Works in HD
Dapagliflozin Benefits Patients with FSGS, Study Shows
Photo by Robina Weermeijer on Unsplash

Dapagliflozin Benefits Patients with FSGS, Study Shows

  • Post author:Jessica Lynn
  • Post published:June 16, 2021
  • Post category:Focal segmental glomerulosclerosis

From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was held fully online. However, that…

Continue Reading Dapagliflozin Benefits Patients with FSGS, Study Shows
All Patients Enrolled in TMB-001 Trial for Congenital Ichthyosis
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All Patients Enrolled in TMB-001 Trial for Congenital Ichthyosis

  • Post author:Jessica Lynn
  • Post published:June 16, 2021
  • Post category:Ichthyosis

In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…

Continue Reading All Patients Enrolled in TMB-001 Trial for Congenital Ichthyosis
JSP191 for SCID Granted Rare Pediatric Disease Designation
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JSP191 for SCID Granted Rare Pediatric Disease Designation

  • Post author:Jessica Lynn
  • Post published:June 15, 2021
  • Post category:Severe Combined Immunodeficiency

Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…

Continue Reading JSP191 for SCID Granted Rare Pediatric Disease Designation
FDA Approves Ryplazim for Hypoplasminogenemia
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FDA Approves Ryplazim for Hypoplasminogenemia

  • Post author:Jessica Lynn
  • Post published:June 15, 2021
  • Post category:Hypoplasminogenemia or Type 1 Plasminogen Deficiency

Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…

Continue Reading FDA Approves Ryplazim for Hypoplasminogenemia
Iptacopan for IgAN Meets Phase 2 Trial Primary Endpoint
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Iptacopan for IgAN Meets Phase 2 Trial Primary Endpoint

  • Post author:Jessica Lynn
  • Post published:June 14, 2021
  • Post category:IgA Nephropathy/IgA Nephropathy (IgAN)

From June 5-8, 2021, the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress. Though the event was held virtually, the meeting still expanded…

Continue Reading Iptacopan for IgAN Meets Phase 2 Trial Primary Endpoint
Positive Topline Data Available on Itolizumab for GvHD
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Positive Topline Data Available on Itolizumab for GvHD

  • Post author:Jessica Lynn
  • Post published:June 14, 2021
  • Post category:Graft versus Host Disease

According to a June 11 press release from biotechnology company Equillium, Inc. ("Equillium"), positive topline data is now available from the Phase 1b EQUATE clinical trial. Within the clinical trial,…

Continue Reading Positive Topline Data Available on Itolizumab for GvHD
New Safety and Tolerability Data Available on CAEL-101 for AL Amyloidosis
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New Safety and Tolerability Data Available on CAEL-101 for AL Amyloidosis

  • Post author:Jessica Lynn
  • Post published:June 14, 2021
  • Post category:Amyloidosis

Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…

Continue Reading New Safety and Tolerability Data Available on CAEL-101 for AL Amyloidosis
New Data Available on Oxbryta for SCD
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New Data Available on Oxbryta for SCD

  • Post author:Jessica Lynn
  • Post published:June 14, 2021
  • Post category:Sickle Cell Disease

Currently, the European Hematology Association (EHA) is holding its 2021 Virtual Congress through June 17, 2021. During the event, researchers are expounding on new insights and research within the hematological…

Continue Reading New Data Available on Oxbryta for SCD
PREVNAR 20 for Multiple IPD Serotypes Now FDA-Approved
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PREVNAR 20 for Multiple IPD Serotypes Now FDA-Approved

  • Post author:Jessica Lynn
  • Post published:June 14, 2021
  • Post category:Invasive pneumococcal disease

On June 8, 2021, pharmaceutical and biotechnology company Pfizer Inc. ("Pfizer") shared that its Pneumococcal 20-valent Conjugate Vaccine (PREVNAR 20) is now FDA-approved. The vaccine helps protect against 20 different…

Continue Reading PREVNAR 20 for Multiple IPD Serotypes Now FDA-Approved
Ultomiris Now FDA Approved for Pediatric Patients with PNH
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Ultomiris Now FDA Approved for Pediatric Patients with PNH

  • Post author:Jessica Lynn
  • Post published:June 10, 2021
  • Post category:paroxysmal nocturnal hemoglobinuria

As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…

Continue Reading Ultomiris Now FDA Approved for Pediatric Patients with PNH
SPG302 for ALS Receives Orphan Drug Designation
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SPG302 for ALS Receives Orphan Drug Designation

  • Post author:Jessica Lynn
  • Post published:June 10, 2021
  • Post category:Amyotrophic Lateral Sclerosis

  In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…

Continue Reading SPG302 for ALS Receives Orphan Drug Designation
Secukinumab Effective for Treating JIA
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Secukinumab Effective for Treating JIA

  • Post author:Jessica Lynn
  • Post published:June 9, 2021
  • Post category:Juvenile idiopathic arthritis

From June 2-5, 2021, the European Alliance of Associations for Rheumatology (EULAR) held its EULAR 2021 Virtual Congress. During the event, participants discussed basic, translational, and clinical science within the…

Continue Reading Secukinumab Effective for Treating JIA
Etrasimod Granted Orphan Drug Status for EoE
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Etrasimod Granted Orphan Drug Status for EoE

  • Post author:Jessica Lynn
  • Post published:June 9, 2021
  • Post category:Eosinophilic Esophagitis

According to a recent news release from biopharmaceutical company Arena Pharmaceuticals, Inc. ("Arena"), the company's investigational therapy, etrasimod, received Orphan Drug designation from the FDA. Currently, Arena is evaluating etrasimod…

Continue Reading Etrasimod Granted Orphan Drug Status for EoE
Study: Oral Octreotide Provides Positive Results in Acromegaly Patients
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Study: Oral Octreotide Provides Positive Results in Acromegaly Patients

  • Post author:Kendall Mason
  • Post published:June 8, 2021
  • Post category:Acromegaly

A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…

Continue Reading Study: Oral Octreotide Provides Positive Results in Acromegaly Patients
Add-On Voclosporin for Better LN Patient Outcomes
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Add-On Voclosporin for Better LN Patient Outcomes

  • Post author:Jessica Lynn
  • Post published:June 7, 2021
  • Post category:Lupus Nephritis

Early on in 2021, the FDA approved voclosporin as an add-on treatment for patients with lupus nephritis (LN), a frequent complication of lupus. Ultimately, this approval was a positive decision,…

Continue Reading Add-On Voclosporin for Better LN Patient Outcomes
Bylvay Earns Positive CHMP Opinion for PFIC
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Bylvay Earns Positive CHMP Opinion for PFIC

  • Post author:Jessica Lynn
  • Post published:June 4, 2021
  • Post category:Progressive Familial Intrahepatic Cholestasis

  In a recent news release on Morningstar, biopharmaceutical company Albireo Pharma, Inc. ("Albireo") shared that its investigational drug candidate Bylvay (odevixibat) earned a positive opinion from the European Medicines…

Continue Reading Bylvay Earns Positive CHMP Opinion for PFIC
CTI-1601 for FRDA On Hold After Monkey Study Deaths
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CTI-1601 for FRDA On Hold After Monkey Study Deaths

  • Post author:Jessica Lynn
  • Post published:June 4, 2021
  • Post category:Friedreich ataxia

  Sometimes, medical studies just don't go the way that companies want them to. According to MedCity News, this is what biotechnology company Larimar Therapeutics ("Larimar") was faced with during…

Continue Reading CTI-1601 for FRDA On Hold After Monkey Study Deaths
FDA Clears LION-101 IND for LGMD21/R9
PhotoLizM / Pixabay

FDA Clears LION-101 IND for LGMD21/R9

  • Post author:Jessica Lynn
  • Post published:June 1, 2021
  • Post category:Limb-girdle muscular dystrophy

In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…

Continue Reading FDA Clears LION-101 IND for LGMD21/R9
Additional Data Available on RGX-121 for MPS II
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Additional Data Available on RGX-121 for MPS II

  • Post author:Jessica Lynn
  • Post published:May 27, 2021
  • Post category:Hunter Syndrome/MPS II/MPS II/MPS II (Hunter Syndrome)/Mucopolysaccharidosis

From May 11-14, 2021, the American Society of Gene and Cell Therapy (ASGCT) held its 24th Annual Meeting. Due to COVID-19, the meeting was held virtually - but that didn't…

Continue Reading Additional Data Available on RGX-121 for MPS II
Cotoretigene Toliparvovec for XLRP Fails in Clinical Trial
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Cotoretigene Toliparvovec for XLRP Fails in Clinical Trial

  • Post author:Jessica Lynn
  • Post published:May 26, 2021
  • Post category:Retinitis pigmentosa

While some therapies offer potential benefits to patients, not all therapies show efficacy within the clinical trial environment. Recently, shares MedCityNews, this occurred with biotechnology company Biogen Inc.'s ("Biogen") gene…

Continue Reading Cotoretigene Toliparvovec for XLRP Fails in Clinical Trial
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Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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