Alnylam Submits a Clinical Trial Authorization Application for Their NASH Treatment
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Alnylam Submits a Clinical Trial Authorization Application for Their NASH Treatment

Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH).…

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MK-6482 Receives Breakthrough Therapy Designation for VHL-Related RCC
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MK-6482 Receives Breakthrough Therapy Designation for VHL-Related RCC

  According to Ryan McDonald of Cure Today, the FDA recently granted Breakthrough Therapy Designation to MK-6482, an investigational HIF-2α inhibitor developed by Merck. The orally-administered drug, designed to treat…

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ICYMI: SM-88 Receives Orphan Drug Designation as Pancreatic Cancer Treatment
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ICYMI: SM-88 Receives Orphan Drug Designation as Pancreatic Cancer Treatment

  Earlier this week, biotechnology company Time Technologies announced that its drug candidate SM-88 received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. The FDA defines…

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Ovid Therapeutics and Columbia University are Working on a Treatment for KIF1A Associated Neurological Disorder

According to a story from GlobeNewswire, the biopharmaceutical company Ovid Therapeutics Inc. and Columbia University have recently entered a collaborative partnership that will be focused on developing gene-based treatments for…

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A Possible Treatment for Pancreatic Cancer Just Earned Orphan Drug Designation
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A Possible Treatment for Pancreatic Cancer Just Earned Orphan Drug Designation

According to a story from BioSpace, the biotechnology company Tyme, Inc. has just announced that it has earned Orphan Drug designation for its experimental product candidate SM-88. SM-88, also known…

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This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions
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This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions

  A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…

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Duchenne Muscular Dystrophy: Two Patients with a Rare Mutation Were Just Dosed with Gene Therapy
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Duchenne Muscular Dystrophy: Two Patients with a Rare Mutation Were Just Dosed with Gene Therapy

According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…

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Tuberous Sclerosis Complex: CBD Oral Solution Epidiolex Approved to Treat Seizures
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Tuberous Sclerosis Complex: CBD Oral Solution Epidiolex Approved to Treat Seizures

According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated…

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Rare Pediatric Disease Designation Given for Investigative Lymphatic Malformations Therapy
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Rare Pediatric Disease Designation Given for Investigative Lymphatic Malformations Therapy

Rare Pediatric Disease Designation has just been granted by the FDA for an investigative therapy for lymphatic malformations (LMs). This therapy is called TARA-002 and was developed by Protara Therapeutics.…

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OWEAR Makes Datasets and Software More Accessible to Aid Research Technology
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OWEAR Makes Datasets and Software More Accessible to Aid Research Technology

By Danielle Bradshaw from In The Cloud Copy Shimmer Research has announced that OWEAR - the Open Wearables Initiative - has decided to make their health technologies, like their database…

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Moebius Syndrome Patient Explains How to Emote While Wearing a Mask
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Moebius Syndrome Patient Explains How to Emote While Wearing a Mask

Moebius Syndrome Moebius syndrome is a rare condition that causes an inability to move the eyes laterally and facial paralysis. A similar type of paralysis can also occur as a…

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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress
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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress

Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…

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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation
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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation

According to a story from PR Newswire, the biotechnology company INOVIO has just announced that the company's investigational product candidate INO-3107 has earned Orphan Drug designation from the US Food…

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FDA-Approved iOS App Coordinates Neuromodulation Therapies
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FDA-Approved iOS App Coordinates Neuromodulation Therapies

  According to Parkinson's News Today, the FDA recently approved an iOS app to assist patients with neurological disorders. Developed by Abbott, the Patient Controller App offers unique telemedicine at…

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Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing
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Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing

Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…

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New Study Evaluates Peripheral Neuropathy Associated with Chemotherapy in Charcot-Marie-Tooth Disease Gene Variants
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New Study Evaluates Peripheral Neuropathy Associated with Chemotherapy in Charcot-Marie-Tooth Disease Gene Variants

Chemotherapy-induced peripheral neuropathy, or CIPN, is a common side effect of chemotherapy. A recent study aimed to uncover whether or not oxaliplatin chemotherapy increased risk of CIPN for those with…

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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising
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X-Linked Retinitis Pigmentosa Investigative Gene Therapy is Looking Promising

Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…

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