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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Musculoskeletal

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Drugs

September 2020

Tofacitinib for Ankylosing Spondylitis

The active substance in tofacitinib works by blocking the action of enzymes (proteins) known as Janus kinases. These enzymes play an important role in the process of inflammation that occurs in rheumatoid, psoriatic arthritis and ulcerative colitis. By blocking the enzymes’ action, tofacitinib helps reduce the inflammation and other symptoms of these diseases. Tofacitinib is administered orally. If licensed, tofacitinib would offer an additional treatment option for patients with active AS, who have responded inadequately to conventional therapy.

Drugs

August 2020

Edasalonexent for Duchene muscular dystrophy

Edasalonexent is currently in clinical development for the treatment of male paediatric patients with duchenne muscular dystrophy (DMD). DMD is a rare genetic condition caused by a mistake or abnormality in a gene called dystrophin, located on the X chromosome (one of the two sex chromosomes). Chromosomes are tiny structures inside cells made from deoxyribonucleic acid (DNA) and proteins. Males have one X chromosome, while females have two X chromosomes. As males have one X chromosome, DMD is much more common in males. DMD is fatal condition with no cure. It causes progressive muscle weakness and often leads to loss of walking ability by the age of twelve, as well as problems with the heart and lungs. Current treatment options only target a particular mistake or abnormality and focus on slowing progression of the disease.

Drugs

May 2020

Vosoritide for achondroplasia

Vosoritide is in clinical development for the treatment of achondroplasia. Achondroplasia represents the most common form of short-limb dwarfism, a condition where the bones in the arms and legs do not form properly and are shorter than normal. Patients with achondroplasia have a short stature, an enlarged head with a prominent forehead, bowed legs, ear problems, respiratory issues, compression of the spinal cord, as well as short fingers, toes, lower legs and upper arms. No pharmacologic therapies have been approved for achondroplasia in the EU.

Drugs

February 2020

Garetosmab for preventing abnormal bone formation in fibrodysplasia ossificans progressiva

Garetosmab is in clinical development for the prevention of abnormal bone formation, outside of the normal skeleton (heterotopic ossification), and soft-tissue flare-ups in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a very rare disease caused by a mutation in the gene ACVR1. The mutation results in the formation of unwanted bone in muscles, tendons, and ligaments throughout the body. Abnormal, misplaced and mis-shapen (heterotopic) bone can bridge across joints causing immobility, scoliosis, and other deformities. Patients usually require a wheelchair by the time they reach their 20s. Death often results in the 40s from complications, such as pneumonia, heart failure and loss of mobility in the chest, neck and jaw.

Drugs

November 2019

Palovarotene for flare-ups in fibrodysplasia ossificans progressiva – first line

Palovarotene is in clinical development for episodic use in the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a disabling condition, caused by the formation of bony bars within the muscles of the body (HO). This bone formation is usually first noticed in early childhood as a series of hard lumps in the neck or along the spine. These lumps, which may be tender, gradually shrink in size as the affected muscles are replaced by bone. The appearance of bony lumps in muscles is usually spontaneous but can also be provoked by any injury to the muscles.

Drugs

November 2019

Palovarotene for episodic and chronic use in fibrodysplasia ossificans progressiva – first line

Palovarotene is in clinical development for episodic and chronic use in the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a disabling condition, caused by the formation of bony bars within the muscles of the body (HO). This bone formation is usually first noticed in early childhood as a series of hard lumps in the neck or along the spine. These lumps, which may be tender, gradually shrink in size as the affected muscles are replaced by bone. The appearance of bony lumps in muscles is usually spontaneous but can also be provoked by any injury to the muscles.

Drugs

October 2017

Tocilizumab (RoACTEMRA) [subcutaneous injection with an auto injector device] for adult patients with moderate to severe active rheumatoid arthritis – after DMARD failure

There is currently no cure for rheumatoid arthritis. The most common treatment options are steroids to reduce inflammation, medications to reduce pain and inflammation and medications that slow the progression of joint damage from RA. Tocilizumab is already licensed for the treatment of RA as both intravenous and subcutaneous (pre-filled syringe) formulations, however its use, …

Drugs

May 2017

Tendoncel for the treatment of tennis elbow

Tendoncel is a gel which can be applied directly onto the skin of the elbow. Tendoncel gel contains a mixture of growth factors (a small substance which encourages cells to grow) which help the tendon to repair and heal. A clinical trial of tendoncel in people with severe tennis elbow showed improvements in elbow pain.If …

Drugs

May 2017

Romosozumab for osteoporosis in men

Romosozumab arose from a genetic discovery that revealed the body’s own natural ability to increase bone strength. It is a treatment which aims to block the activity of the protein sclerostin. This diminishes bone breakdown and removal and stimulates bone formation, thereby increasing bone strength. The effectiveness and safety of romosozumab for the treatment of …

Drugs

April 2017

Romosozumab for postmenopausal osteoporosis

Romosozumab arose from a genetic discovery that revealed the body’s own natural ability to increase bone strength. It is a treatment which aims to block the activity of the protein sclerostin. This diminishes bone breakdown and removal and stimulates bone formation, thereby increasing bone strength. The effectiveness and safety of romosozumab in women with postmenopausal …

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