Glycogen

• Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary CD71 Centyrin – Gys1 siRNA conjugate ABX1100, for the treatment of Pompe Disease.
• The presentations will take place at the WORLD Symposium 2024 in San Diego, CA, from February 4-6, 2024.
• “We are excited for the opportunity to share our promising data with the Lysosomal Storage Disease and Pompe communities,” said Steven Nadler, Ph.D., Senior Vice President of Translation Research of Aro Biotherapeutics.
• Following are additional details about the conferences and presentations:
  Title: Nonclinical studies in non-human primates on ABX1100: A Centyrin – siRNA conjugate for the treatment of Pompe disease

• ABX1100 is a first-in-class molecule representing a new approach to reducing toxic levels of glycogen that build up in muscles of patients with Pompe Disease.
• “We are thrilled to advance ABX1100 into the clinic,” said Mittie Doyle, MD, FACR, Chief Medical Officer of Aro Biotherapeutics.
• The study is designed to assess safety, tolerability, pharmacokinetics along with various pharmacodynamic biomarkers that will be evaluated to demonstrate target engagement.
• The company anticipates presenting data from the Phase 1 study in 2024.

• ELK GROVE VILLAGE, Ill., Sept. 28, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy has been selected by Amicus Therapeutics to dispense Pombiliti™ (cipaglucosidase alfa-atga) and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.
• Please see full Prescribing Information, including BOXED WARNING, for POMBILITI and full Prescribing Information for OPFOLDA , also available at amicusrx.com .
• Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase.
• We are pleased to partner with Amicus to bring Pombiliti and Opfolda to patients suffering from this devastating disease.

• When you go on a diet, you don’t just lose fat – you lose muscle too.
• To lose weight (body fat), you need to be in a calorie deficit.
• During the first few days in a calorie deficit, the body uses up its small reservoir of glycogen stores for energy.
• Once the glycogen stores are used up, the body shifts to metabolising fat to get the energy it needs to function.

Muscle loss

• Muscle loss will also happen regardless of whether you lose weight gradually or quickly.
• This is debatable, with research showing people lose as much muscle on high-protein weight loss diets as people who followed other types of diets.
• But studies comparing different types of diets have found that low-fat high-carb diets seem to offer the same, if not better, fat loss than low-carb, high-fat diets – with no differences in muscle loss.

Protein and exercise

• Given all that has been said, the only way to prevent muscle loss somewhat while losing weight is to combine exercise (particularly resistance exercise and endurance exercise) with a diet higher in protein.
• This is because exercise stimulates muscle growth – but this process can only happen if you have an adequate supply of protein.
• It’s suggested adults normally aim to consume 0.8g of protein per kilogram of body weight per day to maintain muscle mass.
• But given the extra demand exercise places on the muscles, a person will probably need to consume 1.2-1.5g of protein per kilogram of body weight to preserve muscle during weight loss.