Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

March 9, 2020May 15, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

February 21, 2020February 21, 2020

SRK-015 Data Continue to Support Potential for SMA, Scholar Rock Says

News

Preclinical and clinical data on SRK-015 continue to support the therapy’s potential to treat spinal muscular atrophy (SMA), developer Scholar Rock said in a press release. Recruitment for a ... Read more

November 15, 2019November 17, 2019

New Screening Kit Quickly and Thoroughly Analyzes SMN1, SMN2 Genes in Lab, Asuragen Says

News

Asuragen has launched the AmplideX PCR/CE SMN1/2 Plus Kit, which according to the company provides the most comprehensive analysis of genes associated with spinal muscular atrophy (SMA) on the ... Read more

November 8, 2019November 17, 2019

Prenatal Blood Test for SMA Now Available in Four Countries in Europe, BillionToOne Announces

News

BillionToOne’s non-invasive prenatal blood test, which helps diagnose hereditary and rare diseases in fetuses, is now available in Germany, Austria, Switzerland, and the Netherlands, the company announced. The test, ... Read more

July 15, 2019August 20, 2019

Prenatal Blood Test for SMA and Other Single-Gene Diseases Now Available, BillionToOne Announces

News

The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary and rare diseases in fetuses. Called UNITY, the ... Read more

July 10, 2019November 19, 2019

Adding Reldesemtiv to Spinraza or Investigational SMN-C1 Improved Muscle Function in SMA Mouse Model

News

Adding reldesemtiv to survival of motor neuron (SMN) restoration therapies — namely Spinraza (nusinersen) or the investigational SMN-C1 (an analogue to risdiplam) — can improve muscle function in a ... Read more

January 11, 2019February 4, 2019

Many Parents Believe Healthcare for Children with SMA Needs Improvement, Study Reports

News

Optimal care of children with spinal muscular atrophy (SMA) requires improving competence and an adequate exchange of knowledge among healthcare professionals, as well maintaining a close dialogue with the ... Read more

December 21, 2018January 4, 2019

Risdiplam Granted EMA’s PRIME Designation for Potential in Spinal Muscular Atrophy

News

The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of ... Read more

November 14, 2018November 20, 2018

New Technique, MUNIX, May Capture Motor Neuron Loss and Muscle Status in Type 3 and 4 Adults

News

A non-invasive electrophysiology technique called motor unit number index, or MUNIX, can effectively assess the loss of motor nerve cells and muscle impairment in adults with spinal muscular atrophy ... Read more

August 6, 2018August 8, 2018

Mizzou Researchers Launch Shift Pharmaceuticals to Test Potential SMA Therapy

News

Researchers at Mizzou — the University of Missouri — have started a new company, Shift Pharmaceuticals, to test the clinical effectiveness of a molecule that may help improve the ... Read more

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