FAST: Foundation for Angelman Syndrome Therapeutics | Strategic Alliance Partners

Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.

Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.

The FDA recognized the potential of proCase, which is being developed to treat blood clots and does not increase the risk of bleeding.

The FDA has granted a fast track designation to miransertib for the treatment of PIK3CA-Related Overgrowth Spectrum, a group of ultra-rare genetic disorders.

The FDA has granted a fast track designation to pamrevlumab for the treatment of patients with idiopathic pulmonary fibrosis (IPF).

The FDA has granted a fast track designation to Immune Pharmaceuticals, Inc.’s bertilimumab for the treatment of bullous pemphigoid.

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.

The FDA has granted a fast track designation to FCX-013, a gene therapy for the treatment of patients with moderate to severe localized scleroderma.

The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.

The FDA has granted a fast track designation to gene therapy product candidate AAV-CNGB3 for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

Diacerein 1% ointment (CCP-020) received a fast track designation from the FDA for the treatment of epidermolysis bullosa simplex.

The FDA has granted a fast track designation to Nohla Therapeutics’ dilanubicel (NLA101) for patients with high-risk hematologic malignancies receiving an allogeneic cord blood transplant.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

The FDA has granted a fast track designation to diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome.

The US Food and Drug Administration (FDA) has granted fast track designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

The FDA has granted Fast Track Designation for Cyprium Therapeutics’ CUTX-101, developed for the treatment of certain patients with classic Menkes disease.

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) novel small molecule therapy, AS2015, has been granted SBIR Fast-Track grant.

The FDA granted Fast Track designation to REGENXBIO Inc for its product, RGX-111, a novel, single-dose investigational treatment for mucopolysaccharidosis type 1.

Harmony Biosciences, LLC, announced that the FDA has granted Breakthrough Therapy and Fast Track designations to its product, pitolisant, for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy.

The FDA granted GeneTx Biotherapeutics LLC orphan-drug designation for GTX-101, the company’s potential treatment for Angelman syndrome.

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A new meta-analysis of 102 randomized controlled diet trials found that consumption of polyunsaturated fat instead of carbohydrate or saturated fat reduces blood sugar levels and insulin resistance while also increasing the body’s ability to make insulin.

Following a diet that mirrors the habits of fasting can reduce the symptoms of multiple sclerosis.

The overwhelming experience of preparing for colonoscopies may no longer have to be tolerated on an empty stomach.

You’ve heard it time and time again: eat breakfast. But this practice may be particularly beneficial in people with type 2 diabetes – but not just any breakfast.

The incidence and mortality of pancreatic adenocarcinoma, the fourth leading cause of cancer deaths in the United States, are both on the rise. Globally, the disease is responsible for 227,000 deaths annually. Researchers from the National Taiwan University College of Medicine recently published a meta-analysis that demonstrated a dose-response relationship between fasting blood glucose levels and pancreatic cancer risk. They also examined the link between prediabetes and pancreatic cancer risk.

The American Diabetes Association revised Standards of Medical Care for patients with diabetes include several changes in recommendations regarding testing and treatment goals.

Children who eat low-sugar cereals for breakfast tend to make smarter nutritional choices than those who start the day with sweet-filled meals.

Drug for diabetic neuropathic pain receives FDA fast track designation.