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Home » Topics » Disease categories and therapies » Musculoskeletal

Musculoskeletal
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Inozyme’s INZ-701 shows promise for rare disease ENPP1 deficiency

May 15, 2025
By Karen Carey
No Comments
Just two months after Inozyme Pharma Inc. cut its workforce by 25% and prioritized activities to focus on a BLA filing for INZ-701 for ENPP1 deficiency, interim phase III data from its Energy 3 trial showed consistent safety and immunogenicity and increased phosphate levels in patients treated with the rare disease enzyme replacement therapy.
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Apimeds Pharmaceuticals raises $13.5M from NYSE American debut

May 14, 2025
By Marian (YoonJee) Chu
No Comments
Apimeds Pharmaceuticals US Inc. (APUS), a subsidiary of Kospi-listed Inscobee Inc., said May 12 that it raised $13.5 million from its stock sale on the NYSE American exchange May 9 through an offering of 3.375 million shares at $4 per share. Hopewell, N.J.-based APUS is the second pharmaceutical company with Asian ties to float shares on the U.S. market this year.
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Art concept for inflamed human tissue
Immune

Kymera unveils new oral IRF5 degrader for immuno-inflammatory diseases

May 12, 2025
No Comments
Kymera Therapeutics Inc. has announced a new oral IRF5 degrader program with potential to treat immuno-inflammatory diseases, such as lupus, Sjögren’s syndrome, rheumatoid arthritis and inflammatory bowel disease.
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3D illustration of a liposome
Neurology/psychiatric

Ahead Therapeutics advances myasthenia gravis program toward toxicology studies

May 9, 2025
No Comments
Ahead Therapeutics SL has received positive feedback from the EMA on its way toward initiating regulatory toxicology studies for its lead program in myasthenia gravis. The feedback supports the company’s scientific approach.
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Approved stamp

South Korea approves record number of biosimilars in 2024

May 6, 2025
By Marian (YoonJee) Chu
South Korea’s Ministry of Food and Drug Safety approved 18 biosimilar products in 2024, making it a record year for domestic biosimilar approvals since the agency’s first nod of Celltrion Inc.’s Remsima, a reference product of Remicade (infliximab), in 2012.
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Illustration showing cross section of skeletal muscle
Musculoskeletal

Lack of NAD does not alter muscle function or accelerate aging

May 5, 2025
By Mar de Miguel
No Comments
Adult skeletal muscle tolerates a lack of the coenzyme nicotinamide adenine dinucleotide (NAD), according to a study led by scientists at the University of Copenhagen. Their results suggest that adverse effects previously associated with congenital NAD depletion in this tissue may be due to impaired muscle development rather than to a deficiency of this molecule.
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Approved stamp

South Korea approves record number of biosimilars in 2024

April 30, 2025
By Marian (YoonJee) Chu
South Korea’s Ministry of Food and Drug Safety approved 18 biosimilar products in 2024, making it a record year for domestic biosimilar approvals since the agency’s first nod of Celltrion Inc.’s Remsima, a reference product of Remicade (infliximab), in 2012.
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Vaccination
Musculoskeletal

New tolerogenic antigen-specific vaccine boosts VISTA+ Tregs and prevents arthritis in RA model

April 29, 2025
Rheumatoid arthritis (RA) is a chronic autoimmune disorder marked by joint inflammation, cartilage loss and bone damage. Although biological disease-modifying antirheumatic drugs have improved treatment outcomes, the disease remains incurable.
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Musculoskeletal

Shanghai Qilu Pharmaceutical discovers new immunoproteasome inhibitors

April 28, 2025
Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. has described immunoproteasome inhibitors reported to be useful for the treatment of systemic lupus erythematosus and arthritis.
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Illustration of motor neuron connecting to muscle fiber

‘Complement’-ary approaches? FcRn holds ground in myasthenia gravis

April 21, 2025
By Randy Osborne
Watchers of the percolating myasthenia gravis space are waiting eagerly for data from Dianthus Therapeutics Inc.’s phase II Magic study testing DNTH-103, an active C1s inhibitor, compared to placebo in patients with anti-AChR-positive generalized disease.
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